☆ 4.7 Review

Catching Them Early: Framework Parameters and Progress for Prenatal and Childhood Application of Advanced Therapies

PHARMACEUTICS (2022)

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Article Biochemistry & Molecular Biology

Engineered virus-like particles for efficient in vivo delivery of therapeutic proteins

Samagya Banskota et al.

Summary: We have developed and applied engineered DNA-free virus-like particles (eVLPs) for efficient delivery of gene editing tools, achieving high levels of base editing. By overcoming the limitations of cargo packaging, release, and localization, the fourth-generation eVLPs demonstrated efficient base editing in mouse and human cells. In addition, the off-target effects of eVLPs were significantly reduced compared to other delivery methods such as AAV or plasmid.

CELL (2022)

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Article Biotechnology & Applied Microbiology

CD19 or CD20 CAR T Cell Therapy Demonstrates Durable Antitumor Efficacy in Patients with Central Nervous System Lymphoma

Rui Liu et al.

Summary: This study evaluated the clinical efficacy of CD19 or CD20 CAR T cell therapy for patients with central nervous system lymphoma. The results showed that CAR T cell therapy was effective and resulted in complete remission in some patients. The median duration of remission was 22.4 months. Combination therapy and early CAR T cell therapy after relapse may contribute to long-term remission.

HUMAN GENE THERAPY (2022)

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Article Oncology

High efficacy and safety of CD38 and BCMA bispecific CAR-T in relapsed or refractory multiple myeloma

Yuanyan Tang et al.

Summary: The study showed that BCMA-CD38 bispecific CAR-T cell therapy is effective in treating R/R MM patients, with high response rate, low recurrence rate, and manageable cytokine release syndrome (CRS), providing a promising treatment option.

JOURNAL OF EXPERIMENTAL & CLINICAL CANCER RESEARCH (2022)

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Article Medicine, General & Internal

Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1/2 trial and expanded access

Francesca Fumagalli et al.

LANCET (2022)

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Article Oncology

A combination of humanized anti-BCMA and murine anti-CD38 CAR-T cell therapy in patients with relapsed or refractory multiple myeloma

Huan Zhang et al.

Summary: This study evaluated the safety and efficacy of a combination of humanized anti-BCMA and murine anti-CD38 CAR-T cell therapy in patients with relapsed or refractory multiple myeloma. The results showed that the combination therapy has the potential to significantly improve overall response rate and survival rate in R/RMM patients, but may also lead to adverse reactions in some patients.

LEUKEMIA & LYMPHOMA (2022)

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Review Cell & Tissue Engineering

Modified mesenchymal stem cells in cancer therapy: A smart weapon requiring upgrades for wider clinical applications

Carla Vicinanza et al.

Summary: Mesenchymal stem stromal cells (MSC) have the ability to target cancer cells, but their clinical efficacy is limited. Ex vivo cell modifications, such as genetic engineering, membrane modification, and cytokine priming, are needed to improve the anti-cancer properties of MSC.

WORLD JOURNAL OF STEM CELLS (2022)

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Article Multidisciplinary Sciences

From hair to liver: emerging application of hair follicle mesenchymal stem cell transplantation reverses liver cirrhosis by blocking the TGF-β/Smad signaling pathway to inhibit pathological HSC activation

Qi Liu et al.

Summary: Hair follicle mesenchymal stem cells (HF-MSCs) have potential therapeutic effects on liver cirrhosis (LC) by improving liver function and ameliorating liver pathology. The effects are possibly achieved by blocking the TGF-13/Smad pathway and inhibiting the pathological activation of hepatic stellate cells (HSCs).

PEERJ (2022)

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Article Oncology

Anti-CAIX BBζ CAR4/8 T cells exhibit superior efficacy in a ccRCC mouse model

Yufei Wang et al.

Summary: Improving CAR-T cell therapy for solid tumors requires a better understanding of CAR design and cellular composition. A study found that CAR-T cells with a CD4/CD8 ratio of BB zeta exhibited superior efficacy in clearing clear-cell renal cell carcinoma (ccRCC) in a mouse model, demonstrating long-term tumor-free outcome.

MOLECULAR THERAPY-ONCOLYTICS (2022)

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Article Hematology

Engraftment Effects after Intra-Bone Marrow versus Intravenous Allogeneic Stem Cell Transplantation in a Reduced-Intensity Conditioning Dog Leukocyte Antigen-Identical Canine Model

Stephanie Schaefer et al.

Summary: The study compared the engraftment efficiency of IBM-HSCT and IV-HSCT following reduced-intensity conditioning in a canine model, finding that engraftment and hematopoietic recovery were comparable regardless of the infusion rate, with only IBM60 group showing greater weight loss compared to the IV group.

TRANSPLANTATION AND CELLULAR THERAPY (2022)

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Review Oncology

Extended Treatment With Mesenchymal Stromal Cells-Frankfurt am Main in a Pediatric Patient With Steroid-refractory Acute Gastrointestinal Graft-Versus-Host Disease: Case Report and Review of the Literature

Bernd Gruhn et al.

Summary: Mesenchymal stromal cells (MSCs) have shown promising results in treating pediatric patients with steroid-refractory acute graft-versus-host disease (GVHD), leading to a high response rate and potential complete remission. The use of MSCs for more than 4 weeks can be considered based on treatment protocols or individual cases, and endoscopic evaluation of aGVHD activity in patients with persistent gastrointestinal symptoms can guide the administration of a second MSC cycle for optimal outcomes. Future research should explore the tissue repair properties of MSCs as a factor in their efficacy for gastrointestinal acute GVHD.

JOURNAL OF PEDIATRIC HEMATOLOGY ONCOLOGY (2021)

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Article Hematology

Uncertainty in an era of transformative therapy for haemophilia: Addressing the unknowns

Glenn F. Pierce

Summary: Haemophilia treatment is entering a new era with gene transfer emerging as an effective long-term option, although there are still many unanswered questions and uncertainties surrounding viral vector-mediated gene transfer biology. The community's acceptance of this uncertainty, along with the need for continued research to address the unknowns, will shape the introduction of gene therapy for haemophilia to a broader patient population in various countries.

HAEMOPHILIA (2021)

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Article Hematology

Targeting a cytokine checkpoint enhances the fitness of armored cord blood CAR-NK cells

May Daher et al.

Summary: The study combined targeting of CIS protein with CAR engineering of NK cells, leading to improved NK cell effector function and successful antitumor activity in a lymphoma mouse model. This approach represents a promising milestone in the development of next-generation NK cells for cancer immunotherapy.

BLOOD (2021)

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Article Cell & Tissue Engineering

Ex vivo-expanded highly pure ABCB5+ mesenchymal stromal cells as Good Manufacturing Practice-compliant autologous advanced therapy medicinal product for clinical use: process validation and first in-human data

Andreas Kerstan et al.

Summary: The study demonstrates the potential of a novel subpopulation of mesenchymal stromal cells called ABCB5(+) MSCs manufactured as ATMPs through a GMP-compliant process, to provide a clinically relevant wound closure strategy for patients with chronic therapy-resistant wounds. The treatment showed a median wound size reduction of 63% at 12 weeks and early relief of pain in patients with therapy-resistant chronic venous ulcers. The data presented contribute to substantiating the idea of using ABCB5(+) MSCs as an effective treatment option for chronic therapy-resistant wounds.

CYTOTHERAPY (2021)

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Review Pharmacology & Pharmacy

Targeted drug delivery for maternal and perinatal health: Challenges and opportunities

Anjali Sharma et al.

Summary: Complications during pregnancy can have harmful effects on maternal and fetal health, necessitating targeted treatment strategies. Emerging research demonstrates intricate signaling between maternal and fetal cells providing opportunities for interventions.

ADVANCED DRUG DELIVERY REVIEWS (2021)

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Article Engineering, Biomedical

PLGA-Nanoparticles for Intracellular Delivery of the CRISPR-Complex to Elevate Fetal Globin Expression in Erythroid Cells

Luis J. Cruz et al.

Summary: The development of CRISPR/Cas9-PLGA-NPs has allowed for efficient gene editing of HSPCs without causing cellular toxicity. This technology provides a promising tool for in vivo treatment of hemoglobinopathies and other genetic diseases.

BIOMATERIALS (2021)

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Review Pharmacology & Pharmacy

Developing cell therapies as drug products

Rachele Ciccocioppo et al.

Summary: In the last 20 years, global regulatory frameworks have been addressing the challenges posed by using cellular products as new therapeutic tools. Advanced Therapy Medicinal Products have shown potential in changing the natural course of diseases like cancers, opportunistic infections, and chronic inflammatory conditions. Over 50 cell-based products have obtained market authorization globally, highlighting the increasing importance of developing cells as innovative therapeutic vehicles.

BRITISH JOURNAL OF PHARMACOLOGY (2021)

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Article Biochemistry & Molecular Biology

HIV in pregnancy: Mother-to-child transmission, pharmacotherapy, and toxicity

Lukas Cerveny et al.

Summary: HIV infection has adverse effects on pregnancy outcomes, necessitating antiretroviral treatment for pregnant women to reduce mother-to-child transmission. However, uncertainties remain regarding changes in drug metabolism during pregnancy and the long-term effects of antiretrovirals on fetuses.

BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR BASIS OF DISEASE (2021)

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Meeting Abstract Hematology

Safety and Efficacy Profile of Autologous CD30.CAR-T-Cell Therapy in Patients with Relapsed or Refractory Classical Hodgkin Lymphoma (CHARIOT Trial)

Sairah Ahmed et al.

BLOOD (2021)

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Meeting Abstract Hematology

Posoleucel (ALVR105), an Off-the-Shelf, Multivirus-Specific T-Cell Therapy, for the Prevention of Viral Infections Post-HCT: Results from an Open-Label Cohort of a Phase 2 Trial

Sanjeet S. Dadwal et al.

BLOOD (2021)

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Meeting Abstract Hematology

A Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Interim Results

Donald B. Kohn et al.

BLOOD (2021)

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Article Cell & Tissue Engineering

Comparison of regulatory pathways for the approval of advanced therapies in the European Union and the United States

Carolina Iglesias-Lopez et al.

Summary: Regulatory agencies in the European Union (EU) and in the United States of America (USA) have adapted and launched regulatory pathways to accelerate patient access to innovative therapies, such as advanced therapy medicinal products (ATMPs). The main regulatory milestones reached by the approved ATMPs are similar in both regions, with the exception of the time for MAA evaluation, the number of authorized products in the regions, and the type of authorization for some products.

CYTOTHERAPY (2021)

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Article Hematology

Anti-CD117 immunotherapy to eliminate hematopoietic and leukemia stem cells

Norman F. Russkamp et al.

Summary: Immunologic conditioning for hematopoietic stem cell transplantation is a promising approach to target diseases while minimizing collateral damage and inflammation to other organs. New technologies and agents are bringing this goal closer to reality, with the potential for more effective and selective treatment.

EXPERIMENTAL HEMATOLOGY (2021)

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Article Obstetrics & Gynecology

Nanoparticles in pregnancy: the next frontier in reproductive therapeutics

Natasha Pritchard et al.

Summary: Nanomedicine technology in reproductive medicine and obstetrics holds promise for targeted therapeutic strategies that enhance efficacy and minimize off-target effects. Through nanoparticle delivery systems, interventions can effectively address various issues in reproductive medicine, providing new treatment options for women's health and reproductive capacity.

HUMAN REPRODUCTION UPDATE (2021)

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Review Biotechnology & Applied Microbiology

Engineering precision nanoparticles for drug delivery

Michael J. Mitchell et al.

Summary: The development of nanoparticles has shown promising clinical applications, overcoming the limitations of therapeutic interventions and biological barriers. Precision therapies have enhanced treatment efficacy through personalized interventions. Advancements in nanoparticle design aim to optimize drug delivery, improving efficacy in general applications and enabling tailored designs for precision applications.

NATURE REVIEWS DRUG DISCOVERY (2021)

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Review Biochemistry & Molecular Biology

Mesenchymal Stem Cell: A Friend or Foe in Anti-Tumor Immunity

Carl Randall Harrell et al.

Summary: Mesenchymal stem cells (MSCs) play a crucial role in regulating the interactions between immune cells and tumor cells, affecting tumor growth and immune responses. Their plasticity allows them to adapt to the tumor microenvironment and potentially be used as therapeutic agents in cancer immunotherapy.

INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2021)

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Article Multidisciplinary Sciences

Ionizable lipid nanoparticles for in utero mRNA delivery

Rachel S. Riley et al.

Summary: Using ionizable lipid nanoparticles, mRNA can be effectively and safely delivered to mouse fetuses to produce therapeutic secreted proteins, providing a platform for prenatal gene editing and protein replacement.

SCIENCE ADVANCES (2021)

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Review Pharmacology & Pharmacy

The Neonatal and Juvenile Pig in Pediatric Drug Discovery and Development

Miriam Ayuso et al.

Summary: The use of neonatal and juvenile animal models, particularly pigs, in pediatric drug discovery and development shows promising potential for assessing drug safety and understanding disease mechanisms. However, limitations and unexplored aspects of this large animal model still need to be addressed for further development of nonclinical safety models for pediatric drug development.

PHARMACEUTICS (2021)

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Article Engineering, Biomedical

Effects of recipient age, heparin release and allogeneic bone marrow-derived stromal cells on vascular graft remodeling

Richard Johnson et al.

Summary: This study evaluated the impacts of allogeneic bone marrow-derived stromal cells and recipient age on the long-term remodeling of vascular grafts. The results showed significant effects of these factors on graft outcomes. Additionally, compared to acellular grafts, cellular grafts showed efficient recruitment of vascular cells to form more organized structures.

ACTA BIOMATERIALIA (2021)

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Review Pharmacology & Pharmacy

Delivery technologies for in utero gene therapy

Rohan Palanki et al.

Summary: Advances in prenatal imaging, molecular diagnostic tools, and genetic screening have made it possible to treat congenital diseases before clinical symptoms appear. In utero gene therapy shows promise in correcting a wide range of genetic disorders within the womb, but faces challenges in biocompatibility and intracellular delivery efficiency of transgenes. Further research is needed to develop novel delivery modalities for clinical translation of this therapeutic approach.

ADVANCED DRUG DELIVERY REVIEWS (2021)

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Review Pharmacology & Pharmacy

Environmental risk assessment of advanced therapies containing genetically modified organisms in the EU

Rhys Whomsley et al.

Summary: The European Union has implemented a streamlined procedure for assessing the environmental risks of gene therapy medicinal products, aiming to remove hurdles for developers and facilitate faster access to patients.

BRITISH JOURNAL OF CLINICAL PHARMACOLOGY (2021)

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Article Medicine, General & Internal

Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome

B. Gentner et al.

Summary: Hematopoietic gene therapy for Hurler syndrome in patients resulted in extensive metabolic correction in both peripheral tissues and the central nervous system, showing promising treatment outcomes.

NEW ENGLAND JOURNAL OF MEDICINE (2021)

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Review Genetics & Heredity

Evolving AAV-delivered therapeutics towards ultimate cures

Xiangjun He et al.

Summary: In recent years, gene therapy has entered a new era with AAV emerging as a potent vector for gene transfer in vivo. AAV-based gene therapy products have been approved by FDA and EMA for the treatment of previously incurable diseases, marking significant progress in the field. The combination of AAV vectors with genome editing tools has introduced new therapeutic modalities, expanding treatment options for untreatable diseases.

JOURNAL OF MOLECULAR MEDICINE-JMM (2021)

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Review Biotechnology & Applied Microbiology

Update on Clinical Ex Vivo Hematopoietic Stem Cell Gene Therapy for Inherited Monogenic Diseases

Francesca Tucci et al.

Summary: Gene transfer into autologous HSPCs shows potential in treating monogenic blood disorders and metabolic diseases. Two products based on HSPCs have been approved in the EU, with more expected in the near future. Despite achievements, challenges remain for HSPC-GT.

MOLECULAR THERAPY (2021)

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Review Cell & Tissue Engineering

Human umbilical cord-derived mesenchymal stem/stromal cells: a promising candidate for the development of advanced therapy medicinal products

Miryam Mebarki et al.

Summary: UC-MSCs are considered as a promising perspective for treating immune and inflammatory diseases due to their immunomodulatory and anti-inflammatory properties. The classification of UC-MSC-based products as advanced therapy medicinal products in Europe poses challenges for stakeholders in terms of adaptation from cell banks to manufacturing medicines. Despite the potential, the development of UC-MSC-based ATMPs remains a real challenge for both academic institutions and pharmaceutical companies.

STEM CELL RESEARCH & THERAPY (2021)

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Article Oncology

CD19 and CD30 CAR T-Cell Immunotherapy for High-Risk Classical Hodgkin's Lymphoma

YuanBo Xue et al.

Summary: The study indicated that combination therapy with anti-CD19 and CD30 CAR T-cells may be safe and effective for relapsed/refractory classical Hodgkin's Lymphoma patients. Only a small percentage of patients were found eligible for this treatment, but those who received it showed promising outcomes without severe adverse events. Further clinical trials are needed to explore the potential of CD19 and CD30 CAR T-cell combination therapy.

FRONTIERS IN ONCOLOGY (2021)

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Review Pharmacology & Pharmacy

Advanced Therapy Medicinal Products for the Eye: Definitions and Regulatory Framework

Marina Lopez-Paniagua et al.

Summary: ATMPs are innovative and complex biological products for human use, including cell therapy, tissue engineering, gene therapy, and combined products with medical devices. The development of ATMPs for treating eye diseases has rapidly expanded in recent years, offering novel therapeutic approaches for incurable conditions.

PHARMACEUTICS (2021)

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Article Hematology

In vivo HSPC gene therapy with base editors allows for efficient reactivation of fetal gamma-globin in beta-YAC mice

Chang Li et al.

Summary: Base editors delivered via HDAd vectors efficiently induced gamma-globin reactivation in human erythroid progenitor cells, offering a promising strategy for the treatment of beta-hemoglobinopathies. Important advantages of base editing over CRISPR/Cas9 include lower rates of off-target deletions and absence of detectable toxicity in human CD34(+) cells.

BLOOD ADVANCES (2021)

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Article Hematology

The cost-effectiveness of gene therapy for severe hemophilia B: a microsimulation study from the United States perspective

Nancy S. Bolous et al.

Summary: This study compared the cost-effectiveness of AAV-mediated gene therapy for severe hemophilia B in the United States with traditional treatments, and found that gene therapy was more cost-effective at a threshold of $150,000/quality-adjusted life-year. The study also conducted sensitivity analyses and found that gene therapy remained cost-effective in certain scenarios, indicating its potential as a treatment option for patients with severe hemophilia B.

BLOOD (2021)

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Article Pharmacology & Pharmacy

Health Technology Assessment of Advanced Therapy Medicinal Products: Comparison Among 3 European Countries

Lucia Gozzo et al.

Summary: Despite being centrally approved, there are differences among European countries in terms of health technology assessment (HTA), particularly when it comes to evaluating the therapeutic value of ATMPs. While there is often disagreement on clinical assessment, access to ATMPs is usually guaranteed with varying timing and limitations.

FRONTIERS IN PHARMACOLOGY (2021)

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Article Medicine, Research & Experimental

mRNA-engineered mesenchymal stromal cells expressing CXCR2 enhances cell migration and improves recovery in IBD

Qiaojia Li et al.

Summary: In the treatment of inflammatory bowel diseases, modifying MSCs with specific chemokine receptors through mRNA engineering can enhance their homing ability and demonstrate high efficacy and anti-inflammatory effects.

MOLECULAR THERAPY-NUCLEIC ACIDS (2021)

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Review Immunology

Aging of the Hematopoietic Stem Cell Niche: New Tools to Answer an Old Question

Francesca Matteini et al.

Summary: The aging of the bone marrow niche leads to declining HSC function, but certain niche structures and signals are crucial for maintaining HSC function. The use of new technical tools has revealed the impact of BM niche aging on HSCs.

FRONTIERS IN IMMUNOLOGY (2021)

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Editorial Material Biotechnology & Applied Microbiology

Call for More Effective Regulation of Clinical Trials with Advanced Therapy Medicinal Products Consisting of or Containing Genetically Modified Organisms in the European Union

Stuart Beattie

Summary: Compliance with GMO requirements for ATMPs in EU Member States varies significantly, leading to delays in clinical trials and making it difficult to conduct multicenter trials. The European Commission should consider simplifying regulations to improve access to gene therapies for European patients.

HUMAN GENE THERAPY (2021)

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Article Oncology

Off-the-shelf Vδ1 gamma delta T cells engineered with glypican-3 (GPC-3)-specific chimeric antigen receptor (CAR) and soluble IL-15 display robust antitumor efficacy against hepatocellular carcinoma

Amani Makkouk et al.

Summary: The use of engineered V delta 1 CAR T cells targeting GPC-3 in HCC shows promising efficacy and can overcome challenges faced by traditional T cell therapies in solid tumors.

JOURNAL FOR IMMUNOTHERAPY OF CANCER (2021)

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Article Cell Biology

Nucleofection of Adipose Mesenchymal Stem/Stromal Cells: Improved Transfection Efficiency for GMP Grade Applications

Francesco Agostini et al.

Summary: NF is a safe and non-viral transfection method for ASC, and improving NF efficiency is crucial for clinical applications. Using SRGF for ASC expansion led to increased NF efficiency and altered cellular functions. A statistical correlation between NF efficiency and lipid raft availability was observed, suggesting potential mechanisms underlying SRGF impact on ASC transfection efficiency.

CELLS (2021)

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Article Cell Biology

Comparison of Three CD3-Specific Separation Methods Leading to Labeled and Label-Free T Cells

Ronald Weiss et al.

Summary: T cells, crucial in immune response, have been studied for cancer treatment. Different isolation methods were used to obtain T cells in large quantities with high viability and purity. While MACS(R) labels the cells, TACS(R) and REAlease(R) separate cells without labeling, with similar functionality post isolation.

CELLS (2021)

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Review Cell Biology

Development of Stem Cell-Derived Immune Cells for Off-the-Shelf Cancer Immunotherapies

Yan-Ruide Li et al.

Summary: Cell-based cancer immunotherapy has revolutionized the treatment of hematological malignancies, with CAR-T cell therapies achieving unprecedented clinical response rates. The autologous nature of current CAR-T cell products poses challenges, while allogeneic cell products face concerns such as GvHD and allorejection. Advances in developing stem cell-engineered allogeneic cell therapies aim to overcome these limitations.

CELLS (2021)

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Review Pharmacology & Pharmacy

Medical Device Development for Children and Young People-Reviewing the Challenges and Opportunities

Paul Dimitri et al.

Summary: The article emphasizes the importance of developing customized medical devices for children and young people and highlights the huge potential of the global pediatric healthcare market. Collaboration across multiple disciplines is necessary to ensure successful development of medical devices targeting this population.

PHARMACEUTICS (2021)

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Review Chemistry, Multidisciplinary

BBB-crossing adeno-associated virus vector: An excellent gene delivery tool for CNS disease treatment

Wenli Chen et al.

Summary: This review discusses various approaches to improve transportation of therapeutic agents to the CNS, focusing on the advantages of using BBB-crossing AAVs as gene delivery vectors and the different types of BBB-AAV vectors that have been developed for potential applications in CNS diseases.

JOURNAL OF CONTROLLED RELEASE (2021)

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Review Cell & Tissue Engineering

A review and update for registered clinical studies of stem cells for non-tumorous and non-hematological diseases

Jianhua Chen et al.

Summary: This study provided consultative information on potential indications of stem cells in the treatment of non-tumorous and non-hematopoietic system conditions by screening clinical trials. The analysis included various diseases, with motor system diseases and central nervous system diseases being the most studied, and common cell types used include MSCs, BM-SCs, etc.

REGENERATIVE THERAPY (2021)

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Article Oncology

Human umbilical cord mesenchymal stem cells ameliorate acute liver failure by inhibiting apoptosis, inflammation and pyroptosis

Mengting Liu et al.

Summary: The study demonstrated that UC-MSCs have therapeutic effects on acute liver failure by inhibiting apoptosis, inflammation, and pyroptosis. UC-MSCs significantly reduced mortality, improved liver function, inhibited inflammatory responses, and attenuated cell apoptosis and pyroptosis in the liver tissue. Additionally, UC-MSCs suppressed inflammatory cell infiltration in liver tissue.

ANNALS OF TRANSLATIONAL MEDICINE (2021)

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Review Cell & Tissue Engineering

Mesenchymal stem cells and extracellular vesicles in therapy against kidney diseases

Yuling Huang et al.

Summary: MSC-EVs have demonstrated significant therapeutic effects on acute kidney injury, chronic kidney disease, diabetic nephropathy, and atherosclerotic renovascular disease, while their roles in transplanted kidneys remain controversial.

STEM CELL RESEARCH & THERAPY (2021)

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Article Cell & Tissue Engineering

Stem cells for bronchopulmonary dysplasia in preterm infants: A randomized controlled phase II trial

So Yoon Ahn et al.

Summary: The study demonstrated the safety and feasibility of MSC transplantation for BPD in preterm infants through a phase I clinical trial. The efficacy of MSCs for BPD treatment in premature infants was investigated in a phase II trial, where although inflammatory cytokines were significantly reduced, the overall primary outcome was not significantly improved. Subgroup analysis showed a significant improvement with MSC transplantation in the 23-24 GW group.

STEM CELLS TRANSLATIONAL MEDICINE (2021)

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Article Multidisciplinary Sciences

Delivery of the Cas9/sgRNA Ribonucleoprotein Complex in Immortalized and Primary Cells via Virus-like Particles (Nanoblades)

Philippe E. Mangeot et al.

Summary: The CRISPR-Cas system has brought democratization of genome editing in eukaryotic cells, but the delivery of Cas9 protein and sgRNA into target cells remains a technical challenge. Nanoblades, based on MLV, have been developed to overcome issues with traditional viral vectors and can efficiently deliver Cas9 protein and guide RNA without the need for a coding transgene.

JOVE-JOURNAL OF VISUALIZED EXPERIMENTS (2021)

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Review Pharmacology & Pharmacy

Drug Safety in Translational Paediatric Research: Practical Points to Consider for Paediatric Safety Profiling and Protocol Development: A Scoping Review

Beate Aurich et al.

Summary: Translational paediatric drug development involves the exchange between basic, clinical, and population-based research to enhance children's health. This scoping review aimed to search and summarize literature for practical guidance on establishing a paediatric safety specification and integrating it into a paediatric protocol.

PHARMACEUTICS (2021)

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Review Cell Biology

Translational Animal Models Provide Insight Into Mesenchymal Stromal Cell (MSC) Secretome Therapy

Rebecca M. Harman et al.

Summary: The therapeutic potential of MSC secretome is being intensely studied, but its complexity and variability pose challenges for its application in human medicine. A deeper understanding of the factors that make up the secretome and the ability to manipulate MSCs to consistently secrete factors of biologic importance will improve MSC therapy.

FRONTIERS IN CELL AND DEVELOPMENTAL BIOLOGY (2021)

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Review Biochemistry & Molecular Biology

Gene Therapy for Lysosomal Storage Disorders: Ongoing Studies and Clinical Development

Giulia Massaro et al.

Summary: This study provides a comprehensive overview of the current state of gene therapy in lysosomal diseases, including viral gene delivery vectors, relevant pre-clinical studies, and ongoing clinical trials in recent years.

BIOMOLECULES (2021)

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Review Pharmacology & Pharmacy

Non-viral Vectors in Gene Therapy: Recent Development, Challenges, and Prospects

Hui Zu et al.

Summary: Although non-viral vectors in gene therapy have low cytotoxicity and immunogenicity, they still face challenges in gene transfer efficiency, specificity, gene expression duration, and safety. Overcoming these obstacles is necessary to enhance the effectiveness of non-viral vectors in gene therapy.

AAPS JOURNAL (2021)

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Article Hematology

Characterization of HLH-like manifestations as a CRS variant in CD22 CAR T cells

Daniel A. Lichtenstein et al.

Summary: In a subset of patients with cytokine release syndrome (CRS), chimeric antigen receptor (CAR) T-cell toxicities resembling hemophagocytic lymphohistiocytosis (HLH) can occur, known as carHLH. This study comprehensively characterized the manifestations and timing of carHLH, highlighting factors such as preinfusion natural killer cell lymphopenia and higher bone marrow T-cell:NK cell ratio associated with its development. Additionally, carHLH was characterized by persistent elevation of HLH-associated inflammatory cytokines following CAR T-cell expansion.

BLOOD (2021)

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Review Immunology

CAR T cells: Building on the CD19 paradigm

Anat Globerson-Levin et al.

Summary: Therapeutic use of CARs targeting CD19 has led to significant success in treating B-cell malignancies, sparking interest in T-cell engineering and cancer immunotherapy. Advances in CAR design, cell manufacturing, and genome editing hold promise for safer and more effective genetically instructed immunity, with potential applications in a wide range of diseases in the future.

EUROPEAN JOURNAL OF IMMUNOLOGY (2021)

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Review Biochemistry & Molecular Biology

Adipose-Derived Stem Cells in the Treatment of Perianal Fistulas in Crohn's Disease: Rationale, Clinical Results and Perspectives

Etienne Buscail et al.

Summary: In situ injection of ADSCs is effective in treating perianal fistulas in CD patients. ADSCs exert their therapeutic effects through paracrine phenomena and display various beneficial properties such as anti-inflammatory, pro-angiogenic, and immunomodulatory effects. Numerous clinical trials have shown promising results with a high cure rate and safety profile, indicating the potential for future standardized treatment approaches.

INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2021)

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Review Cell & Tissue Engineering

Comparability: what we can learn from the review of advanced therapy medicinal products

Alexis Cockroft et al.

Summary: This study evaluated publicly available summaries and reports to explore regulators' expectations for comparability data in gene and cell therapy products. The research found deficiencies, additional information requests, and major objections related to comparability in some approved products. Post-approval authorization obligations were imposed on six products, highlighting the importance of comparability data in regulatory applications.

REGENERATIVE MEDICINE (2021)

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Editorial Material Biochemistry & Molecular Biology

In Utero Gene Therapy: Progress and Challenges

Sourav K. Bose et al.

Summary: In utero gene therapy has the potential to treat lethal and morbid perinatal diseases before birth, with the fetus being a compelling patient due to its small size, tolerogenic immune system, and dosing efficiency. However, achieving the promise of genetic treatment before birth requires consideration of numerous clinical, social, and institutional factors.

TRENDS IN MOLECULAR MEDICINE (2021)

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Review Medicine, Research & Experimental

Evaluation of animal models of Crohn's disease with anal fistula (Review)

Shuangshuang Lu et al.

Summary: This review summarized the characteristics and experimental methods of commonly used animal models of CD with anal fistula, pointing out the necessity of establishing a suitable animal preclinical model.

EXPERIMENTAL AND THERAPEUTIC MEDICINE (2021)

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Article Multidisciplinary Sciences

In utero adenine base editing corrects multi-organ pathology in a lethal lysosomal storage disease

Sourav K. Bose et al.

Summary: In utero base editing shows potential in correcting disease-causing mutations before birth, improving survival and alleviating symptoms of diseases like MPSI. This approach highlights the feasibility and efficacy of therapeutic base editing in multiple organs before birth, offering promising treatment options for genetic diseases.

NATURE COMMUNICATIONS (2021)

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Review Cell & Tissue Engineering

CAR-engineered NK cells; a promising therapeutic option for treatment of hematological malignancies

Faroogh Marofi et al.

Summary: CAR-NK cells are safer and more efficient than CAR-T cells in treating tumors, especially hematological malignancies. NK cells can be effectively engineered to express CARs with substantial cytotoxic activity against both hematological and solid tumors.

STEM CELL RESEARCH & THERAPY (2021)

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Review Cell & Tissue Engineering

Nonviral genome engineering of natural killer cells

Gabrielle M. Robbins et al.

Summary: NK cells have the potential to be a crucial part of cancer immunotherapy, but face challenges such as in vivo persistence and the tumor microenvironment. Enhancing NK cell resistance to tumor inhibitory signaling through genome engineering may improve their efficacy in cancer treatment.

STEM CELL RESEARCH & THERAPY (2021)

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Review Cell & Tissue Engineering

Comparison of the efficacy of hematopoietic stem cell mobilization regimens: a systematic review and network meta-analysis of preclinical studies

Chengxin Luo et al.

Summary: Through network meta-analysis comparing the efficacy of different HSC mobilization regimens, it was found that regimens including G-CSF plus AMD3100, Me6, EP80031, and ML141 showed promising results in terms of collecting CFCs and LSK cells.

STEM CELL RESEARCH & THERAPY (2021)

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Article Virology

Adenovirus-a blueprint for gene delivery

Urs F. Greber et al.

Summary: The article discusses the mechanisms by which human adenoviruses deliver their double-stranded DNA genome into the cell nucleus, as well as their effects on lytic cell killing, non-lytic persistent infection, or vector gene expression.

CURRENT OPINION IN VIROLOGY (2021)

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Article Cell Biology

MSC Manufacturing for Academic Clinical Trials: From a Clinical-Grade to a Full GMP-Compliant Process

Chantal Lechanteur et al.

Summary: The study outlines the upgrade of an existing clinical-grade MSC manufacturing process to comply with GMP standards, including reorganization of key aspects and validation of a new variant for specific disease treatment. While GMP manufacturing of MSC products is feasible in academic settings, large-scale production for phase III trials would require collaboration with industrial partners.

CELLS (2021)

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Review Cell Biology

Therapy Development by Genome Editing of Hematopoietic Stem Cells

Lola Koniali et al.

Summary: Advances in HSC genome editing as a treatment for inherited disorders have shown significant progress, with their versatility and accuracy driving potential therapeutic approaches for diseases. The focus of research is on improving the efficiency of HSC modification, treatment tolerability, and the distribution and affordability of corresponding therapies.

CELLS (2021)

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Review Oncology

From CAR-T Cells to CAR-NK Cells: A Developing Immunotherapy Method for Hematological Malignancies

Hui Lu et al.

Summary: The approval of CD19 CAR-engineered T cell products in B-cell malignancies is a breakthrough in CAR-T cell immunotherapy. However, limitations such as GVHD and other adverse effects restrict their wider applications. CAR-NK cells, with their unique characteristics, are considered promising candidates for cellular immunotherapy, offering potential off-the-shelf products for immediate clinical use without HLA-matching restrictions. Researchers are shifting focus from CAR-T cells to CAR-NK cells, discussing their advantages and challenges in clinical applications.

FRONTIERS IN ONCOLOGY (2021)

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Article Oncology

Culturing adequate CAR-T cells from less peripheral blood to treat B-cell malignancies

Lu Han et al.

Summary: A novel method for CAR-T cell preparation from small volumes of peripheral blood has been developed, providing an alternative means of CAR-T cell generation for patients ineligible for leukapheresis. This method resulted in successful expansion of CAR-T cells and promising outcomes in patients with B-cell malignancies.

CANCER BIOLOGY & MEDICINE (2021)

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Article Hematology

Reduction of Graft-versus-Host-Disease in NOD.Cg-Prkdcscid Il2rgtm1Wjl/SzJ (NSG) Mice by Cotransplantation of Syngeneic Human Umbilical Cord-Derived Mesenchymal Stromal Cells

Max Hansen et al.

Summary: This study analyzed the potential beneficial effects of syngeneic versus allogenic treatment, culture expansion of MSCs, and various MSC cell doses and time points of MSC transplantation in a murine GVHD model. The results showed that syngeneic treatment, culture-expanded cells, simultaneous administration, and high doses of UC-MSCs were more effective in reducing CD8(+) T cells, protecting mice from weight loss, and prolonging survival. This animal model of GVHD can be used for further detailed studies on the efficacy of MSCs from alternative sources.

TRANSPLANTATION AND CELLULAR THERAPY (2021)

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Article Medicine, General & Internal

Apligraf as an Alternative to Skin Grafting in the Pediatric Population

Morgan Eudy et al.

Summary: Apligraf, a bioengineered living cell construct, is demonstrated as a viable, if not superior, treatment option for full-thickness skin injuries in the pediatric population.

CUREUS JOURNAL OF MEDICAL SCIENCE (2021)

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Article Surgery

Clinical Analysis of Cultured Epidermal Autograft (JACE) Transplantation for Giant Congenital Melanocytic Nevus

Kento Takaya et al.

Summary: This study investigated the postoperative course of GCMN patients undergoing cultured epidermal autograft transplantation, and found that early removal of nevus tissue with the use of a hydrosurgery system can provide good results while reducing complications.

PLASTIC AND RECONSTRUCTIVE SURGERY-GLOBAL OPEN (2021)

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Article Hematology

Reduced volume of red blood cell priming is safe for pediatric patients undergoing therapeutic plasma exchange

Jose Roberto Luzzi et al.

Summary: The study aimed to determine the safety of administering a reduced volume of RBC priming for pediatric patients undergoing TPE compared to the standard volume recommended by the cell separators' manufacturers. Results showed that restricting the volume of RBC priming to less than 150 mL is safe for pediatric patients weighing more than 10Kg and associated with lower rates of transfusion-related adverse reactions.

TRANSFUSION AND APHERESIS SCIENCE (2021)

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Article Health Policy & Services

Price and reimbursement of advanced therapeutic medicinal products in Europe: are assessment and appraisal diverging from expert recommendations?

Virginia Ronco et al.

Summary: The reimbursement status of ATMPs varies across the European Major five (EU5) countries, with CAR-T being reimbursed in all countries. Outcome-based Managed Entry Agreements (MEAs) are extensively used for ATMPs. Extra funds for hospitals managing ATMPs are only provided in Germany, with additional funding in France.

JOURNAL OF PHARMACEUTICAL POLICY AND PRACTICE (2021)

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Review Biotechnology & Applied Microbiology

The Promise and the Hope of Gene Therapy

Eleni Papanikolaou et al.

Summary: Gene therapy, proposed by visionary scientists over 30 years ago, has shown success in treating certain monogenic diseases. With the introduction of CAR-T cell therapies, gene therapy has expanded to treat a broader range of diseases. The integration of gene therapy into everyday clinical practice is expected to be gradual and impactful.

FRONTIERS IN GENOME EDITING (2021)

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Article Cell & Tissue Engineering

Efficacy of Gene Modification in Placenta-Derived Mesenchymal Stem Cells Based on Nonviral Electroporation

Jae Yeon Kim et al.

Summary: Mesenchymal stem cell-based gene therapy for degenerative diseases is effective and safe, but the exact mechanism of action is unclear. Viral-based gene modification is commonly used due to its efficient vehicles, but carries risks such as oncogenes and chromosomal integration. Nonviral gene delivery techniques are cheaper, easier to handle, and safer, although less efficient.

INTERNATIONAL JOURNAL OF STEM CELLS (2021)

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Review Biochemistry & Molecular Biology

Delivering cellular and gene therapies to patients: solutions for realizing the potential of the next generation of medicine

Kris Elverum et al.

GENE THERAPY (2020)

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Review Pharmacology & Pharmacy

Mouse models in hematopoietic stem cell gene therapy and genome editing

Stefan Radtke et al.

BIOCHEMICAL PHARMACOLOGY (2020)

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Review Cell Biology

Immune modulation by mesenchymal stem cells

Wei Jiang et al.

CELL PROLIFERATION (2020)

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Review Cell & Tissue Engineering

Are all stem cells equal? Systematic review, evidence map, and meta-analyses of preclinical stem cell-based therapies for bronchopulmonary dysplasia

Sajit Augustine et al.

STEM CELLS TRANSLATIONAL MEDICINE (2020)

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Article Oncology

ACCELERATE and European Medicines Agency Paediatric Strategy Forum for medicinal product development of checkpoint inhibitors for use in combination therapy in paediatric patients

Andrew D. J. Pearso et al.

EUROPEAN JOURNAL OF CANCER (2020)

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Review Biotechnology & Applied Microbiology

Public Acceptability of Gene Therapy and Gene Editing for Human Use: A Systematic Review

Juliette Delhove et al.

HUMAN GENE THERAPY (2020)

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Review Oncology

B-cell maturation antigen (BCMA) in multiple myeloma: rationale for targeting and current therapeutic approaches

Nina Shah et al.

LEUKEMIA (2020)

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Review Biotechnology & Applied Microbiology

Retroviral Insertional Mutagenesis in Humans: Evidence for Four Genetic Mechanisms Promoting Expansion of Cell Clones

Frederic D. Bushman

MOLECULAR THERAPY (2020)

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Review Medicine, General & Internal

MSC Based Therapies-New Perspectives for the Injured Lung

Judith Behnke et al.

JOURNAL OF CLINICAL MEDICINE (2020)

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Article Pediatrics

Application of protection motivation theory to clinical trial enrolment for pediatric chronic conditions

Stephanie P. Brooks et al.

BMC PEDIATRICS (2020)

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Review Audiology & Speech-Language Pathology

Fetal gene therapy and pharmacotherapy to treat congenital hearing loss and vestibular dysfunction

Michelle L. Hastings et al.

HEARING RESEARCH (2020)

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Article Biotechnology & Applied Microbiology

Dose Range Finding Studies with Two RPGR Transgenes in a Canine Model of X-Linked Retinitis Pigmentosa Treated with Subretinal Gene Therapy

Chunjuan Song et al.

HUMAN GENE THERAPY (2020)

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Review Immunology

Mechanisms of Fetal T Cell Tolerance and Immune Regulation

Elze Rackaityte et al.

FRONTIERS IN IMMUNOLOGY (2020)

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Review Veterinary Sciences

Canine Models of Inherited Musculoskeletal and Neurodegenerative Diseases

Brett D. Story et al.

FRONTIERS IN VETERINARY SCIENCE (2020)

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Article Cell Biology

Persistent DNA Damage and Senescence in the Placenta Impacts Developmental Outcomes of Embryos

Vijay Pratap Singh et al.

DEVELOPMENTAL CELL (2020)

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Article Biotechnology & Applied Microbiology

Gene Therapy Targeting the Inner Retina Rescues the Retinal Phenotype in a Mouse Model of CLN3 Batten Disease

Sophia-Martha Kleine Holthaus et al.

HUMAN GENE THERAPY (2020)

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Article Pharmacology & Pharmacy

Short and long term impacts of COVID-19 on the pharmaceutical sector

Nayyereh Ayati et al.

DARU-JOURNAL OF PHARMACEUTICAL SCIENCES (2020)

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Article Biochemistry & Molecular Biology

Bispecific anti-CD20, anti-CD19 CAR T cells for relapsed B cell malignancies: a phase 1 dose escalation and expansion trial

Nirav N. Shah et al.

NATURE MEDICINE (2020)

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Article Chemistry, Physical

Risk Management Framework for Nano-Biomaterials Used in Medical Devices and Advanced Therapy Medicinal Products

Elisa Giubilato et al.

MATERIALS (2020)

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Article Materials Science, Biomaterials

Nanoparticle-Mediated Simultaneous Downregulation of Placental Nrf2 and sFlt1 Improves Maternal and Fetal Outcomes in a Preeclampsia Mouse Model

Lei Li et al.

ACS BIOMATERIALS SCIENCE & ENGINEERING (2020)

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Review Medicine, General & Internal

CAR-NK cells: A promising cellular immunotherapy for cancer

Guozhu Xie et al.

EBIOMEDICINE (2020)

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Meeting Abstract Hematology

In Utero Stem Cell Transplantation in Patients with Alpha Thalassemia Major: Interim Results of a Phase 1 Clinical Trial

Tippi C. MacKenzie et al.

BLOOD (2020)

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Meeting Abstract Hematology

A Phase 1/2 Study of Lentiviral-Mediated Ex-Vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Results from Phase 1

Donald B. Kohn et al.

BLOOD (2020)

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Editorial Material Economics

Advanced therapy medicinal products: value judgement and ethical evaluation in health technology assessment

Elisabete Goncalves

EUROPEAN JOURNAL OF HEALTH ECONOMICS (2020)

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Article Multidisciplinary Sciences

Electroporation outperforms in vivo-jetPEI for intratumoral DNA-based reporter gene transfer

Liesl Jacobs et al.

SCIENTIFIC REPORTS (2020)

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Review Biotechnology & Applied Microbiology

Gene Editing and Genotoxicity: Targeting the Off-Targets

Georges Blattner et al.

FRONTIERS IN GENOME EDITING (2020)

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Article Cell & Tissue Engineering

Spatio-Temporal Metabolokinetics and Efficacy of Human Placenta-Derived Mesenchymal Stem/Stromal Cells on Mice with Refractory Crohn's-like Enterocutaneous Fistula

Huixing Hou et al.

STEM CELL REVIEWS AND REPORTS (2020)

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Article Biotechnology & Applied Microbiology

An Approach towards a GMP Compliant In-Vitro Expansion of Human Adipose Stem Cells for Autologous Therapies

Valentin Jossen et al.

BIOENGINEERING-BASEL (2020)

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Review Materials Science, Biomaterials

Design of nanomaterials for applications in maternal/fetal medicine

N'dea S. Irvin-Choy et al.

JOURNAL OF MATERIALS CHEMISTRY B (2020)

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Article Cell & Tissue Engineering

Engineered scaffolds based on mesenchymal stem cells/preosteoclasts extracellular matrix promote bone regeneration

Rui Dong et al.

JOURNAL OF TISSUE ENGINEERING (2020)

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Editorial Material Medicine, Research & Experimental

Innovation and competition in advanced therapy medicinal products

Enrique Seoane-Vazquez et al.

EMBO MOLECULAR MEDICINE (2019)

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Editorial Material Biotechnology & Applied Microbiology

In Utero Gene Therapy Consensus Statement from the IFeTIS

Graca Almeida-Porada et al.

MOLECULAR THERAPY (2019)

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Article Health Care Sciences & Services

Value-Based Pricing for Emerging Gene Therapies: The Economic Case for a Higher Cost-Effectiveness Threshold

Louis P. Garrison et al.

JOURNAL OF MANAGED CARE & SPECIALTY PHARMACY (2019)

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Review Cell Biology

Ex Vivo Expansion of Hematopoietic Stem Cells for Therapeutic Purposes: Lessons from Development and the Niche

Parisa Tajer et al.

CELLS (2019)

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Letter Hematology

Correction of IVS I-110(G>A) β-thalassemia by CRISPR/Cas- and TALEN-mediated disruption of aberrant regulatory elements in human hematopoietic stem and progenitor cells

Petros Patsali et al.

HAEMATOLOGICA (2019)

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Article Pediatrics

Safety of Intratracheal Administration of Human Umbilical Cord Blood Derived Mesenchymal Stromal Cells in Extremely Low Birth Weight Preterm Infants

Steven B. Powell et al.

JOURNAL OF PEDIATRICS (2019)

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Article Genetics & Heredity

Rare Opportunities: CRISPR/Cas-Based Therapy Development for Rare Genetic Diseases

Panayiota Papasavva et al.

MOLECULAR DIAGNOSIS & THERAPY (2019)

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Article Medicine, General & Internal

Lentiviral Gene Therapy Combined with Low-Dose Busulfan in Infants with SCID-X1

Ewelina Mamcarz et al.

NEW ENGLAND JOURNAL OF MEDICINE (2019)

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Article Cell Biology

In utero gene editing for monogenic lung disease

Deepthi Alapati et al.

SCIENCE TRANSLATIONAL MEDICINE (2019)

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Article Cell & Tissue Engineering

In Utero Transplantation of Expanded Autologous Amniotic Fluid Stem Cells Results in Long-Term Hematopoietic Engraftment

Stavros P. Loukogeorgakis et al.

STEM CELLS (2019)

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Article Economics

Estimating the Clinical Pipeline of Cell and Gene Therapies and Their Potential Economic Impact on the US Healthcare System

Casey Quinn et al.

VALUE IN HEALTH (2019)

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Review Polymer Science

Polymeric Nanoparticles in Gene Therapy: New Avenues of Design and Optimization for Delivery Applications

Raj Rai et al.

POLYMERS (2019)

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Review Biotechnology & Applied Microbiology

The Landscape of Cellular and Gene Therapy Products: Authorization, Discontinuations, and Cost

Vaishali Shukla et al.

HUMAN GENE THERAPY CLINICAL DEVELOPMENT (2019)

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Article Endocrinology & Metabolism

Aging and lineage allocation changes of bone marrow skeletal (stromal) stem cells

Jan O. Nehlin et al.

BONE (2019)

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Review Cell & Tissue Engineering

Concise Review: Challenges in Clinical Development of Mesenchymal Stromal/Stem Cells

Ilenia Mastrolia et al.

STEM CELLS TRANSLATIONAL MEDICINE (2019)

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Review Immunology

Mesenchymal Stromal Cell Therapeutic Delivery: Translational Challenges to Clinical Application

Henry Caplan et al.

FRONTIERS IN IMMUNOLOGY (2019)

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Article Biotechnology & Applied Microbiology

Generation and Clinical Application of Gene-Modified Autologous Epidermal Sheets in Netherton Syndrome: Lessons Learned from a Phase 1 Trial

Wei-Li Di et al.

HUMAN GENE THERAPY (2019)

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Review Biochemistry & Molecular Biology

Current Strategies to Enhance Adipose Stem Cell Function: An Update

Yoojin Seo et al.

INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2019)

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Review Oncology

CAR T cell therapy: A new era for cancer treatment

Rimjhim Mohanty et al.

ONCOLOGY REPORTS (2019)

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Review Pediatrics

Mesenchymal stem cells for the prevention of bronchopulmonary dysplasia

Fumihiko Namba

PEDIATRICS INTERNATIONAL (2019)

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Meeting Abstract Hematology

FT576: A Novel Multiplexed Engineered Off-the-Shelf Natural Killer Cell Immunotherapy for the Dual-Targeting of CD38 and Bcma for the Treatment of Multiple Myeloma

Ryan Bjordahl et al.

BLOOD (2019)

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Meeting Abstract Hematology

Changing the Natural History of Fanconi Anemia Complementation Group-A with Gene Therapy: Early Results of US Phase I Study of Lentiviral-Mediated Ex-VivoFANCA Gene Insertion in Human Stem and Progenitor Cells

Agnieszka Czechowicz et al.

BLOOD (2019)

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Article Multidisciplinary Sciences

In Utero Gene Therapy (IUGT) Using GLOBE Lentiviral Vector Phenotypically Corrects the Heterozygous Humanised Mouse Model and Its Progress Can Be Monitored Using MRI Techniques

Panicos Shangaris et al.

SCIENTIFIC REPORTS (2019)

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Meeting Abstract Hematology

FT596: Translation of First-of-Kind Multi-Antigen Targeted Off-the-Shelf CAR-NK Cell with Engineered Persistence for the Treatment of B Cell Malignancies

Jode P. Goodridge et al.

BLOOD (2019)

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Meeting Abstract Hematology

Enhanced Transduction Lentivector Gene Therapy for Treatment of Older Patients with X-Linked Severe Combined Immunodeficiency

Suk See De Ravin et al.

BLOOD (2019)

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Article Biochemistry & Molecular Biology

Therapeutic expression of human clotting factors IX and X following adeno-associated viral vector-mediated intrauterine gene transfer in early-gestation fetal macaques

Jerry K. Y. Chan et al.

FASEB JOURNAL (2019)

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Article Biotechnology & Applied Microbiology

Optimization of CRISPR/Cas9 Delivery to Human Hematopoietic Stem and Progenitor Cells for Therapeutic Genomic Rearrangements

Annalisa Lattanzi et al.

MOLECULAR THERAPY (2019)

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Article Biochemistry & Molecular Biology

Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent β-thalassemia

Sarah Marktel et al.

NATURE MEDICINE (2019)

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Editorial Material Medicine, General & Internal

After the Storm - A Responsible Path for Genome Editing

George Q. Daley et al.

NEW ENGLAND JOURNAL OF MEDICINE (2019)

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Article Multidisciplinary Sciences

Genome editing in primary cells and in vivo using viral-derived Nanoblades loaded with Cas9-sgRNA ribonucleoproteins

Philippe E. Mangeot et al.

NATURE COMMUNICATIONS (2019)

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Editorial Material Critical Care Medicine

Cell-based therapies for acute respiratory distress syndrome

Daniel J. Weiss

LANCET RESPIRATORY MEDICINE (2019)

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Article Critical Care Medicine

Treatment with allogeneic mesenchymal stromal cells for moderate to severe acute respiratory distress syndrome (START study): a randomised phase 2a safety trial

Michael A. Matthay et al.

LANCET RESPIRATORY MEDICINE (2019)

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Article Critical Care Medicine

Paediatric acute respiratory distress syndrome incidence and epidemiology (PARDIE): an international, observational study

Robinder G. Khemani et al.

LANCET RESPIRATORY MEDICINE (2019)

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Article Cell Biology

Children and Adults with Refractory Acute Graft-versus-Host Disease Respond to Treatment with the Mesenchymal Stromal Cell Preparation MSC-FFM-Outcome Report of 92 Patients

Halvard Bonig et al.

CELLS (2019)

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Article Cell Biology

The Phenotype and Secretory Activity of Adipose-Derived Mesenchymal Stem Cells (ASCs) of Patients with Rheumatic Diseases

Ewa Kuca-Warnawin et al.

CELLS (2019)

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Article Genetics & Heredity

Controlling CRISPR Through Law: Legal Regimes as Precautionary Principles

Jacob S. Sherkow

CRISPR JOURNAL (2019)

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Article Orthopedics

Matrix-Associated Autologous Chondrocyte Implantation Is an Effective Treatment at Midterm Follow-up in Adolescents and Young Adults

Arnd Hoburg et al.

ORTHOPAEDIC JOURNAL OF SPORTS MEDICINE (2019)

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Article Cell & Tissue Engineering

In Utero Transplantation of Placenta-Derived Mesenchymal Stromal Cells for Potential Fetal Treatment of Hemophilia A

Priyadarsini Kumar et al.

CELL TRANSPLANTATION (2018)

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Article Cell & Tissue Engineering

A decade of marketing approval of gene and cell-based therapies in the United States, European Union and Japan: An evaluation of regulatory decision-making

D. G. M. Coppens et al.

CYTOTHERAPY (2018)

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Article Cell & Tissue Engineering

Extracellular vesicles released from mesenchymal stromal cells stimulate bone growth in osteogenesis imperfecta

Satoru Otsuru et al.

CYTOTHERAPY (2018)

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Article Hematology

A comparison of intrauterine hemopoietic cell transplantation and lentiviral gene transfer for the correction of severe β-thalassemia in a HbbTh3/+ murine model

Niraja M. Dighe et al.

EXPERIMENTAL HEMATOLOGY (2018)

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Article Biotechnology & Applied Microbiology

Adeno-Associated Virus Gene Therapy in a Sheep Model of Tay-Sachs Disease

Heather L. Gray-Edwards et al.

HUMAN GENE THERAPY (2018)

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Article Biotechnology & Applied Microbiology

Efficient Non-viral Gene Delivery into Human Hematopoietic Stem Cells by Minicircle Sleeping Beauty Transposon Vectors

Marta Holstein et al.

MOLECULAR THERAPY (2018)

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Article Biochemistry & Molecular Biology

Fetal gene therapy for neurodegenerative disease of infants

Giulia Massaro et al.

NATURE MEDICINE (2018)

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Article Medicine, General & Internal

Prenatal Correction of X-Linked Hypohidrotic Ectodermal Dysplasia

Holm Schneider et al.

NEW ENGLAND JOURNAL OF MEDICINE (2018)

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Article Medicine, General & Internal

Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia

A. A. Thompson et al.

NEW ENGLAND JOURNAL OF MEDICINE (2018)

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Review Chemistry, Multidisciplinary

The Placental Barrier: the Gate and the Fate in Drug Distribution

Nino Tetro et al.

PHARMACEUTICAL RESEARCH (2018)

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Article Cell & Tissue Engineering

Long-Term Hematopoietic Engraftment of Congenic Amniotic Fluid Stem Cells After in Utero Intraperitoneal Transplantation to Immune Competent Mice

Panicos Shangaris et al.

STEM CELLS AND DEVELOPMENT (2018)

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Article Multidisciplinary Sciences

In utero nanoparticle delivery for site-specific genome editing

Adele S. Ricciardi et al.

NATURE COMMUNICATIONS (2018)

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Article Medicine, Research & Experimental

Integrating HDAd5/35++ Vectors as a New Platform for HSC Gene Therapy of Hemoglobinopathies

Chang Li et al.

MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2018)

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Article Biochemistry & Molecular Biology

Trophoblast: The central unit of fetal growth, protection and programming

Frantisek Staud et al.

INTERNATIONAL JOURNAL OF BIOCHEMISTRY & CELL BIOLOGY (2018)

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Article Pharmacology & Pharmacy

The Path to Perfect Pediatric Posology - Drug Development in Pediatrics

Joan M. Korth-Bradley

JOURNAL OF CLINICAL PHARMACOLOGY (2018)

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Article Cell & Tissue Engineering

Cell, tissue and gene products with marketing authorization in 2018 worldwide

Natividad Cuende et al.

CYTOTHERAPY (2018)

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Editorial Material Multidisciplinary Sciences

The fix is in utero

Sarah DeWeerdt

NATURE (2018)

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Article Biochemistry & Molecular Biology

In utero CRISPR-mediated therapeutic editing of metabolic genes

Avery C. Rossidis et al.

NATURE MEDICINE (2018)

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Article Medicine, Research & Experimental

Challenges in Advanced Therapy Medicinal Product Development: A Survey among Companies in Europe

Renske M. T. ten Ham et al.

MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2018)

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Review Pediatrics

The Therapeutic Potential of Stem Cells for Bronchopulmonary Dysplasia: It's About Time or Not so Fast ?

Mong Tieng Ee et al.

CURRENT PEDIATRIC REVIEWS (2018)

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Article Oncology

In Vivo Hematopoietic Stem Cell Transduction

Maximilian Richter et al.

HEMATOLOGY-ONCOLOGY CLINICS OF NORTH AMERICA (2017)

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Article Pediatrics

Two-Year Follow-Up Outcomes of Premature Infants Enrolled in the Phase I Trial of Mesenchymal Stem Cells Transplantation for Bronchopulmonary Dysplasia

So Yoon Ahn et al.

JOURNAL OF PEDIATRICS (2017)

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Article Biotechnology & Applied Microbiology

In Utero Transfer of Adeno-Associated Viral Vectors Produces Long-Term Factor IX Levels in a Cynomolgus Macaque Model

Citra N. Z. Mattar et al.

MOLECULAR THERAPY (2017)

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Article Multidisciplinary Sciences

Regeneration of the entire human epidermis using transgenic stem cells

Tobias Hirsch et al.

NATURE (2017)

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Article Medicine, General & Internal

Gene Therapy in a Patient with Sickle Cell Disease

Jean-Antoine Ribeil et al.

NEW ENGLAND JOURNAL OF MEDICINE (2017)

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Review Medicine, General & Internal

Acute Respiratory Distress Syndrome

B. Taylor Thompson et al.

NEW ENGLAND JOURNAL OF MEDICINE (2017)

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Review Ophthalmology

Subretinal Injection: A Review on the Novel Route of Therapeutic Delivery for Vitreoretinal Diseases

Yingqian Peng et al.

OPHTHALMIC RESEARCH (2017)

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Article Genetics & Heredity

Orphan medicinal products in Europe and United States to cover needs of patients with rare diseases: an increased common effort is to be foreseen

Viviana Giannuzzi et al.

ORPHANET JOURNAL OF RARE DISEASES (2017)

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Article Pediatrics

New bronchopulmonary dysplasia and chronic lung disease

Judith A. Voynow

PAEDIATRIC RESPIRATORY REVIEWS (2017)

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Article Pediatrics

Pathophysiology and Management of Acute Respiratory Distress Syndrome in Children

Sabrina M. Heidemann et al.

PEDIATRIC CLINICS OF NORTH AMERICA (2017)

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Article Multidisciplinary Sciences

Intrapancreatic injection of human bone marrow-derived mesenchymal stem/stromal cells alleviates hyperglycemia and modulates the macrophage state in streptozotocin-induced type 1 diabetic mice

Norimitsu Murai et al.

PLOS ONE (2017)

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Review Cell & Tissue Engineering

A roadmap for cost-of-goods planning to guide economic production of cell therapy products

Yonatan Y. Lipsitz et al.

CYTOTHERAPY (2017)

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Article Cell & Tissue Engineering

Suitability of small diagnostic peripheral-blood samples for cell therapy studies

Coralea Stephanou et al.

CYTOTHERAPY (2017)

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Article Pharmacology & Pharmacy

Non-invasive gene targeting to the fetal brain after intravenous administration and transplacental transfer of plasmid DNA using PEGylated immunoliposomes

Eain M. Cornford et al.

JOURNAL OF DRUG TARGETING (2016)

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Article Cell & Tissue Engineering

Generation of a Nonhuman Primate Model of Severe Combined Immunodeficiency Using Highly Efficient Genome Editing

Kenya Sato et al.

CELL STEM CELL (2016)

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Article Hematology

Mesenchymal stromal cells from pooled mononuclear cells of multiple bone marrow donors as rescue therapy in pediatric severe steroid-refractory graft-versus-host disease: a multicenter survey

Zyrafete Kuci et al.

HAEMATOLOGICA (2016)

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Article Medicine, General & Internal

Safety and Wound Outcomes Following Genetically Corrected Autologous Epidermal Grafts in Patients With Recessive Dystrophic Epidermolysis Bullosa

Zurab Siprashvili et al.

JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION (2016)

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Review Medicine, General & Internal

Genome editing in nonhuman primates: approach to generating human disease models

Y. Chen et al.

JOURNAL OF INTERNAL MEDICINE (2016)

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Editorial Material Biotechnology & Applied Microbiology

Timing of Gene Therapy Interventions: The Earlier, the Better

Steven J. Gray

MOLECULAR THERAPY (2016)

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Review Biotechnology & Applied Microbiology

Nanoparticle transport across the placental barrier: pushing the field forward!

Carina Muoth et al.

NANOMEDICINE (2016)

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Article Pediatrics

Cell Therapy for Bronchopulmonary Dysplasia: Promises and Perils

Marius Alexander Moebius et al.

PAEDIATRIC RESPIRATORY REVIEWS (2016)

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Review Genetics & Heredity

Favorable outcomes after in utero transfusion in fetuses with alpha thalassemia major: a case series and review of the literature

Emily M. Kreger et al.

PRENATAL DIAGNOSIS (2016)

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Article Hematology

Comprehensive technical and patient-care optimization in the management of pediatric apheresis for peripheral blood stem cell harvesting

Yoshihiro Ohara et al.

TRANSFUSION AND APHERESIS SCIENCE (2016)

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Article Hematology

Mesenchymal stromal cells from pooled mononuclear cells of multiple bone marrow donors as rescue therapy in pediatric severe steroid-refractory graft-versus-host disease: a multicenter survey

Zyrafete Kuci et al.

HAEMATOLOGICA (2016)

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Article Medicine, Research & Experimental

Functional correction of neurological and somatic disorders at later stages of disease in MPS IIIA mice by systemic scAAV9-hSGSH gene delivery

Haiyan Fu et al.

MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2016)

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Review Medicine, Research & Experimental

In utero stem cell transplantation and gene therapy: rationale, history, and recent advances toward clinical application

Graca Almeida-Porada et al.

MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT (2016)

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Article Multidisciplinary Sciences

Tumor-homing peptides as tools for targeted delivery of payloads to the placenta

Anna King et al.

SCIENCE ADVANCES (2016)

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Review Multidisciplinary Sciences

Efficacy of Mesenchymal Stromal Cell Therapy for Acute Lung Injury in Preclinical Animal Models: A Systematic Review

Lauralyn A. McIntyre et al.

PLOS ONE (2016)

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Review Genetics & Heredity

Lessons Learned from Two Decades of Clinical Trial Experience in Gene Therapy for Fanconi Anemia

Jennifer E. Adair et al.

CURRENT GENE THERAPY (2016)

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Review Biochemistry & Molecular Biology

Human mesenchymal stem cells - current trends and future prospective

Imran Ullah et al.

BIOSCIENCE REPORTS (2015)

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Review Hematology

Xenograft models for normal and malignant stem cells

Susumu Goyama et al.

BLOOD (2015)

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Article Cell & Tissue Engineering

Human Umbilical Cord Mesenchymal Stem Cells: A New Era for Stem Cell Therapy

Dah-Ching Ding et al.

CELL TRANSPLANTATION (2015)

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Article Medicine, General & Internal

T cells expressing CD19 chimeric antigen receptors for acute lymphoblastic leukaemia in children and young adults: a phase 1 dose-escalation trial

Daniel W. Lee et al.

LANCET (2015)

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Article Biotechnology & Applied Microbiology

Long-Term Correction of Sandhoff Disease Following Intravenous Delivery of rAAV9 to Mouse Neonates

Jagdeep S. Walia et al.

MOLECULAR THERAPY (2015)

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Article Critical Care Medicine

Pediatric Acute Respiratory Distress Syndrome: Consensus Recommendations From the Pediatric Acute Lung Injury Consensus Conference

Philippe Jouvet et al.

PEDIATRIC CRITICAL CARE MEDICINE (2015)

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Article Multidisciplinary Sciences

Development of Non-Viral, Trophoblast-Specific Gene Delivery for Placental Therapy

Noura Abd Ellah et al.

PLOS ONE (2015)

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Article Cell & Tissue Engineering

Enrichment of Human Hematopoietic Stem/Progenitor Cells Facilitates Transduction for Stem Cell Gene Therapy

Kismet Baldwin et al.

STEM CELLS (2015)

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Article Hematology

Stem cell mobilization and collection from pediatric patients and healthy children

Musa Karakukcu et al.

TRANSFUSION AND APHERESIS SCIENCE (2015)

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Review Gastroenterology & Hepatology

Challenges in animal modelling of mesenchymal stromal cell therapy for inflammatory bowel disease

Raghavan Chinnadurai et al.

WORLD JOURNAL OF GASTROENTEROLOGY (2015)

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Article Critical Care Medicine

Mesenchymal stem (stromal) cells for treatment of ARDS: a phase 1 clinical trial

Jennifer G. Wilson et al.

LANCET RESPIRATORY MEDICINE (2015)

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Article Medicine, General & Internal

Allogeneic Mesenchymal Stem Cells Restore Endothelial Function in Heart Failure by Stimulating Endothelial Progenitor Cells

Courtney Premer et al.

EBIOMEDICINE (2015)

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Article Cell & Tissue Engineering

Sheep CD34+Amniotic Fluid Cells Have Hematopoietic Potential and Engraft After Autologous In Utero Transplantation

S. W. Steven Shaw et al.

STEM CELLS (2015)

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Review Medicine, General & Internal

Non Viral Vectors in Gene Therapy- An Overview

Murali Ramamoorth et al.

JOURNAL OF CLINICAL AND DIAGNOSTIC RESEARCH (2015)

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Review Pharmacology & Pharmacy

Gene and cell therapy for children - New medicines, new challenges?

Karen F. Buckland et al.

ADVANCED DRUG DELIVERY REVIEWS (2014)

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Article Surgery

Experience of Using Cultured Epithelial Autografts for the Extensive Burn Wounds in Eight Patients

Minoru Hayashi et al.

ANNALS OF PLASTIC SURGERY (2014)

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Article Hematology

Treatment of Graft versus Host Disease with Mesenchymal Stromal Cells: A Phase I Study on 40 Adult and Pediatric Patients

Martino Introna et al.

BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION (2014)

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Article Hematology

Allogeneic Human Mesenchymal Stem Cell Therapy (Remestemcel-L, Prochymal) as a Rescue Agent for Severe Refractory Acute Graft-versus-Host Disease in Pediatric Patients

Joanne Kurtzberg et al.

BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION (2014)

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Article Biotechnology & Applied Microbiology

Allogeneic Cell Therapy Bioprocess Economics and Optimization: Single-Use Cell Expansion Technologies

Ana S. Simaria et al.

BIOTECHNOLOGY AND BIOENGINEERING (2014)

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Article Pediatrics

Mesenchymal Stem Cells for Bronchopulmonary Dysplasia: Phase 1 Dose-Escalation Clinical Trial

Yun Sil Chang et al.

JOURNAL OF PEDIATRICS (2014)

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Article Biotechnology & Applied Microbiology

In Utero Lung Gene Transfer Using Adeno-Associated Viral and Lentiviral Vectors in Mice

Luc Joyeux et al.

HUMAN GENE THERAPY METHODS (2014)

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Review Pharmacology & Pharmacy

In utero therapy for congenital disorders using amniotic fluid stem cells

Durrgah L. Ramachandra et al.

FRONTIERS IN PHARMACOLOGY (2014)

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Article Cell & Tissue Engineering

Actin Cytoskeletal Disruption following Cryopreservation Alters the Biodistribution of Human Mesenchymal Stromal Cells In Vivo

Raghavan Chinnadurai et al.

STEM CELL REPORTS (2014)

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Article Immunology

Preclinical Efficacy and Mechanisms of Mesenchymal Stem Cells in Animal Models of Autoimmune Diseases

Hong Kyung Lee et al.

Immune Network (2014)

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Article Biophysics

Co-transplantation of third-party umbilical cord blood-derived MSCs promotes engraftment in children undergoing unrelated umbilical cord blood transplantation

S. H. Lee et al.

BONE MARROW TRANSPLANTATION (2013)

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Article Endocrinology & Metabolism

Targeted Nanoparticle Delivery of Doxorubicin Into Placental Tissues to Treat Ectopic Pregnancies

Tu'uhevaha J. Kaitu'u-Lino et al.

ENDOCRINOLOGY (2013)

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Article Biotechnology & Applied Microbiology

A Single Intravenous rAAV Injection as Late as P20 Achieves Efficacious and Sustained CNS Gene Therapy in Canavan Mice

Seemin Seher Ahmed et al.

MOLECULAR THERAPY (2013)

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Article Biotechnology & Applied Microbiology

The Fetal Mouse Is a Sensitive Genotoxicity Model That Exposes Lentiviral-associated Mutagenesis Resulting in Liver Oncogenesis

Ali Nowrouzi et al.

MOLECULAR THERAPY (2013)

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Article Biotechnology & Applied Microbiology

Preclinical Safety and Efficacy of Human CD34(+) Cells Transduced With Lentiviral Vector for the Treatment of Wiskott-Aldrich Syndrome

Samantha Scaramuzza et al.

MOLECULAR THERAPY (2013)

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Article Physiology

Bronchioalveolar stem cells increase after mesenchymal stromal cell treatment in a mouse model of bronchopulmonary dysplasia

Kristen A. Tropea et al.

AMERICAN JOURNAL OF PHYSIOLOGY-LUNG CELLULAR AND MOLECULAR PHYSIOLOGY (2012)

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Article Hematology

Risk of complications during hematopoietic stem cell collection in pediatric sibling donors: a prospective European Group for Blood and Marrow Transplantation Pediatric Diseases Working Party study

Jan Styczynski et al.

BLOOD (2012)

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Article Public, Environmental & Occupational Health

The weight of nations: an estimation of adult human biomass

Sarah Catherine Walpole et al.

BMC PUBLIC HEALTH (2012)

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Article Medicine, General & Internal

Cell-based therapies for lung disease

Orquidea Garcia et al.

BRITISH MEDICAL BULLETIN (2012)

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News Item Biotechnology & Applied Microbiology

Gintuit cell therapy approval signals shift at US regulator

Charles Schmidt

NATURE BIOTECHNOLOGY (2012)

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Article Pediatrics

Bone marrow mesenchymal stem cells attenuate lung inflammation of hyperoxic newborn rats

Hongshan Zhang et al.

PEDIATRIC TRANSPLANTATION (2012)

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Editorial Material Respiratory System

Mesenchymal stem cell therapy in acute lung injury: is it time for a clinical trial?

Rob Mac Sweeney et al.

THORAX (2012)

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Article Cardiac & Cardiovascular Systems

Mesenchymal Stem Cell-Mediated Reversal of Bronchopulmonary Dysplasia and Associated Pulmonary Hypertension

Georg Hansmann et al.

Pulmonary Circulation (2012)

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Article Hematology

Efficacy and Safety of Ex Vivo Cultured Adult Human Mesenchymal Stem Cells (Prochymal™) in Pediatric Patients with Severe Refractory Acute Graft-Versus-Host Disease in a Compassionate Use Study

Vinod K. Prasad et al.

BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION (2011)

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Article Biophysics

Co-infusion of ex vivo-expanded, parental MSCs prevents life-threatening acute GVHD, but does not reduce the risk of graft failure in pediatric patients undergoing allogeneic umbilical cord blood transplantation

M. E. Bernardo et al.

BONE MARROW TRANSPLANTATION (2011)

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Article Cardiac & Cardiovascular Systems

Comparison of Transplacental Treatment of Fetal Supraventricular Tachyarrhythmias With Digoxin, Flecainide, and Sotalol Results of a Nonrandomized Multicenter Study

Edgar T. Jaeggi et al.

CIRCULATION (2011)

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Article Multidisciplinary Sciences

A Mouse Model of in Utero Transplantation

Amar Nijagal et al.

JOVE-JOURNAL OF VISUALIZED EXPERIMENTS (2011)

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Article Hematology

Humanized mouse models of Cooley's anemia: correct fetal-to-adult hemoglobin switching, disease onset, and disease pathology

Yongliang Huo et al.

COOLEY'S ANEMIA: NINTH SYMPOSIUM (2010)

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Meeting Abstract Hematology

SINGLE PRE-TRANSPLANT TREATMENT WITH GM-CSF-SECRETING MYELOID LEUKEMIA CELL VACCINE COMBINED WITH AUTOLOGOUS-BMT SIGNIFICANTLY IMPROVES SURVIVAL OF MYELOID LEUKEMIA CHALLENGED MICE

J. Zilberberg et al.

BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION (2010)

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Article Hematology

Platelet-lysate-Expanded Mesenchymal Stromal Cells as a Salvage Therapy for Severe Resistant Graft-versus-Host Disease in a Pediatric Population

Giovanna Lucchini et al.

BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION (2010)

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Review Biochemistry & Molecular Biology

Large animal models of hematopoietic stem cell gene therapy

G. D. Trobridge et al.

GENE THERAPY (2010)

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Article Multidisciplinary Sciences

Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia

Marina Cavazzana-Calvo et al.

NATURE (2010)

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Article Biotechnology & Applied Microbiology

Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN

Kevin D. Foust et al.

NATURE BIOTECHNOLOGY (2010)

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Article Physiology

Hyperoxia disrupts vascular endothelial growth factor-nitric oxide signaling and decreases growth of endothelial colony-forming cells from preterm infants

Hideshi Fujinaga et al.

AMERICAN JOURNAL OF PHYSIOLOGY-LUNG CELLULAR AND MOLECULAR PHYSIOLOGY (2009)

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Article Critical Care Medicine

Bone Marrow Stromal Cells Attenuate Lung Injury in a Murine Model of Neonatal Chronic Lung Disease

Muhammad Aslam et al.

AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE (2009)

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Article Hematology

Preclinical transfusion-dependent humanized mouse model of β thalassemia major

Yongliang Huo et al.

BLOOD (2009)

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Article Biophysics

Transplantation of ex-vivo culture-expanded parental haploidentical mesenchymal stem cells to promote engraftment in pediatric recipients of unrelated donor umbilical cord blood: results of a phase I-II clinical trial

M. L. MacMillan et al.

BONE MARROW TRANSPLANTATION (2009)

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Article Biotechnology & Applied Microbiology

Factors Determining the Risk of Inadvertent Retroviral Transduction of Male Germ Cells After In Utero Gene Transfer in Sheep

Paul J. Park et al.

HUMAN GENE THERAPY (2009)

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Article Biochemistry & Molecular Biology

Humanized Mouse Model of Cooley's Anemia

Yongliang Huo et al.

JOURNAL OF BIOLOGICAL CHEMISTRY (2009)

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Article Biotechnology & Applied Microbiology

Rescue of Pyruvate Kinase Deficiency in Mice by Gene Therapy Using the Human Isoenzyme

Nestor W. Meza et al.

MOLECULAR THERAPY (2009)

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Article Medicine, General & Internal

Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency.

Alessandro Aiuti et al.

NEW ENGLAND JOURNAL OF MEDICINE (2009)

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Article Neurosciences

Survival advantage of neonatal CNS gene transfer for late infantile neuronal ceroid lipofuscinosis

Dolan Sondhi et al.

EXPERIMENTAL NEUROLOGY (2008)

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Review Biochemistry & Molecular Biology

Stem cell-based strategies for treating pediatric disorders of myelin

Steven A. Goldman et al.

HUMAN MOLECULAR GENETICS (2008)

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Article Medicine, General & Internal

Mesenchymal stem cells for treatment of steroid-resistant, severe, acute graft-versus-host disease:: a phase II study

Katarina LeBlanc et al.

LANCET (2008)

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Review Cell & Tissue Engineering

Hematopoietic Stem Cells: Source Matters

Richard L. Haspel et al.

CURRENT STEM CELL RESEARCH & THERAPY (2008)

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Article Biotechnology & Applied Microbiology

Timing of therapeutic intervention determines functional and survival outcomes in a mouse model of late infantile batten disease

Mario A. Cabrera-Salazar et al.

MOLECULAR THERAPY (2007)

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Article Hematology

Hot spots of retroviral integration in human CD34+ hematopoietic cells

Claudia Cattoglio et al.

BLOOD (2007)

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Review Genetics & Heredity

Acute graft versus host disease

David A. Jacobsohn et al.

ORPHANET JOURNAL OF RARE DISEASES (2007)

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Article Biotechnology & Applied Microbiology

Persistent expression of hF.IX after tolerance induction by in utero or neonatal administration of AAV-1-F.IX in hemophilia B mice

Denise E. Sabatino et al.

MOLECULAR THERAPY (2007)

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Article Immunology

Stem cell transplantation: The lung barrier

S. Schrepfer et al.

TRANSPLANTATION PROCEEDINGS (2007)

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Article Biochemistry & Molecular Biology

Correction of junctional epidermolysis bullosa by transplantation of genetically modified epidermal stem cells

Fulvio Mavilio et al.

NATURE MEDICINE (2006)

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Article Hematology

Ex vivo gene therapy with lentiviral vectors rescues adenosine deaminase (ADA)-deficient mice and corrects their immune and metabolic defects

Alessandra Mortellaro et al.

BLOOD (2006)

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Article Pharmacology & Pharmacy

Medicines for children licensed by the European Medicines Agency (EMEA): the balance after 10 years

A. Ceci et al.

EUROPEAN JOURNAL OF CLINICAL PHARMACOLOGY (2006)

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Review Biochemistry & Molecular Biology

Large animal models and gene therapy

M Casal et al.

EUROPEAN JOURNAL OF HUMAN GENETICS (2006)

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Article Biotechnology & Applied Microbiology

Oncogenesis following delivery of a nonprimate lentiviral gene therapy vector to fetal and neonatal mice

M Themis et al.

MOLECULAR THERAPY (2005)

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Article Biochemistry & Molecular Biology

Bone marrow-derived mesenchymal stem cells in repair of the injured lung

M Rojas et al.

AMERICAN JOURNAL OF RESPIRATORY CELL AND MOLECULAR BIOLOGY (2005)

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Article Medicine, General & Internal

Transplantation of umbilical-cord blood in babies with infantile Krabbe's disease

ML Escolar et al.

NEW ENGLAND JOURNAL OF MEDICINE (2005)

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Article Biochemical Research Methods

Targeted gene delivery to telencephalic inhibitory neurons by directional in utero electroporation

V Borrell et al.

JOURNAL OF NEUROSCIENCE METHODS (2005)

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Article Hematology

Human mesenchymal stem cells modulate allogeneic immune cell responses

S Aggarwal et al.

BLOOD (2005)

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Article Biophysics

Human mesenchymal stem cells support unrelated donor hematopoietic stem cells and suppress T-cell activation

B Maitra et al.

BONE MARROW TRANSPLANTATION (2004)

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Article Immunology

A3 adenosine receptor signaling contributes to airway inflammation and mucus production in adenosine deaminase-deficient mice

HWJ Young et al.

JOURNAL OF IMMUNOLOGY (2004)

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Article Cell & Tissue Engineering

Bone tissue engineering for spine fusion: An experimental study on ectopic and orthotopic implants in rats

SM van Gaalen et al.

TISSUE ENGINEERING (2004)

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Article Hematology

Mixed chimerism following in utero hematopoietic stem cell transplantation in murine models of hemoglobinopathy

S Hayashi et al.

EXPERIMENTAL HEMATOLOGY (2003)

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Article Pediatrics

Peripheral blood as a stem cell source for hematopoietic cell transplantation in children: is the effort in vein?

JM Lipton

PEDIATRIC TRANSPLANTATION (2003)

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Article Biochemistry & Molecular Biology

Functional anatomy of siRNAs for mediating efficient RNAi in Drosophila melanogaster embryo lysate

SM Elbashir et al.

EMBO JOURNAL (2001)

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Article Hematology

Cellular immunotherapy of malignancies using the clonal natural killer cell line NK-92

T Tonn et al.

JOURNAL OF HEMATOTHERAPY & STEM CELL RESEARCH (2001)

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