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High-Precision Synthesis of RNA-Loaded Lipid Nanoparticles for Biomedical Applications

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Monkeypox Virus Infection in Humans across 16 Countries - April-June 2022

John P. Thornhill et al.

Summary: This report describes 528 cases of monkeypox in 16 countries. Common clinical findings include rash, mucosal lesions, fever, lethargy, and lymphadenopathy. Few patients required hospitalization.

NEW ENGLAND JOURNAL OF MEDICINE (2022)

Article Multidisciplinary Sciences

Piperazine-derived lipid nanoparticles deliver mRNA to immune cells in vivo

Huanzhen Ni et al.

Summary: This study reports the development of piperazine-containing lipid nanoparticles (Pi-Lipids) that can selectively deliver mRNA to immune cells without the need for targeting ligands. Using in vivo DNA barcoding, the researchers demonstrated that these nanoparticles efficiently delivered mRNA to non-hepatocytes, which has been a challenging task without the use of ligands. The findings highlight the potential of Pi-Lipids and support the hypothesis that lipids with bioactive small-molecule motifs can deliver mRNA in vivo.

NATURE COMMUNICATIONS (2022)

Article Multidisciplinary Sciences

Optimal self-assembly of lipid nanoparticles (LNP) in a ring micromixer

Manon Ripoll et al.

Summary: Lipid nanoparticles (LNPs) have been widely studied for their potential in RNA and DNA delivery, including as mRNA vectors for COVID vaccines. Microfluidics, particularly the ring micromixer, offers advantages for LNP production, such as high mixing quality, low-stress conditions, and fast preparation. This study focuses on the ring micromixer and identifies three regimes that control the mixing performance. Using cryo-TEM and biochemical analysis, the study also shows the correlation between mixing performance and the characteristics of the LNPs produced. The study emphasizes the importance of flow-rate ratio and proposes a physical criterion for producing LNPs with optimal characteristics.

SCIENTIFIC REPORTS (2022)

Review Biotechnology & Applied Microbiology

Recent advances in mRNA-LNP therapeutics: immunological and pharmacological aspects

Seyed Hossein Kiaie et al.

Summary: Over the past decade, the development of messenger RNA therapeutics facilitated by lipid nanoparticles has greatly improved treatment efficacy. However, uncontrolled immune responses and inappropriate pharmacological interventions may limit the effectiveness of mRNA-LNP vaccines. This study investigates the interaction between immune responses and LNP compositions, as well as the pharmacological profiles of mRNA-LNP delivery, providing insights for improving multidisciplinary therapeutics in clinical trials.

JOURNAL OF NANOBIOTECHNOLOGY (2022)

Article Medicine, Research & Experimental

Downregulation of hepatic lipopolysaccharide binding protein improves lipogenesis-induced liver lipid accumulation

Jessica Latorre et al.

Summary: This study found that intervention on liver LBP can reduce liver lipid accumulation and oxidative stress, thus reducing the development of obesity-related fatty liver. However, in other circumstances, liver LBP downregulation does not have an impact on liver steatosis.

MOLECULAR THERAPY-NUCLEIC ACIDS (2022)

Article Public, Environmental & Occupational Health

Monkeypox Outbreak — Nine States, May 2022

Faisal S. Minhaj et al.

MMWR-MORBIDITY AND MORTALITY WEEKLY REPORT (2022)

Article Pharmacology & Pharmacy

The Potential of Cell-Penetrating Peptides for mRNA Delivery to Cancer Cells

Yelee Kim et al.

Summary: In vitro transcribed mRNA has potential in cancer gene therapy and cancer vaccines. Cell-penetrating peptides (CPPs) show promise as gene delivery tools. This study optimized CPP/mRNA complexes and demonstrated their protective effect against RNase, as well as high cellular uptake and protein expression.

PHARMACEUTICS (2022)

Review Oncology

The treatment of advanced non-small cell lung cancer harboring KRAS mutation: a new class of drugs for an old target-a narrative review

Alessia Spagnuolo et al.

Summary: This paper provides an overview of the scientific efforts in KRAS inhibition, highlighting the approval of Sotorasib as the first KRAS G12C inhibitor that offers durable clinical benefit for pre-treated NSCLC patients with KRAS G12C mutation. The research also explores strategies combining KRAS inhibitors with other targeted therapies, chemotherapy, and immunotherapy.

TRANSLATIONAL LUNG CANCER RESEARCH (2022)

Article Chemistry, Multidisciplinary

Membrane-Specific Binding of 4 nm Lipid Nanoparticles Mediated by an Entropy-Driven Interaction Mechanism

Luping Ou et al.

Summary: The interaction between sub 10 nm cationic LNPs and cell membranes is a two-step entropy-driven process, enabling LNPs to differentiate between membranes with different lipid compositions and providing strategies for developing membrane-targeting agents.

ACS NANO (2022)

Article Chemistry, Multidisciplinary

Enzyme-Catalyzed One-Step Synthesis of Ionizable Cationic Lipids for Lipid Nanoparticle-Based mRNA COVID-19 Vaccines

Zhongyu Li et al.

Summary: Ionizable cationic lipid-containing lipid nanoparticles (LNPs) are important non-viral gene delivery platforms, and the developed AA3-DLin LNPs show excellent mRNA delivery efficacy and strong immunogenicity in COVID-19 vaccines.

ACS NANO (2022)

Article Chemistry, Multidisciplinary

Engineering Lipid Nanoparticles for Enhanced Intracellular Delivery of mRNA through Inhalation

Jeonghwan Kim et al.

Summary: By incorporating PEG lipid and beta-sitosterol, the stability and endosomal escape of lipid nanoparticles (LNPs) for lung mRNA therapy could be enhanced, leading to effective treatment.

ACS NANO (2022)

Review Pharmacology & Pharmacy

Microfluidic production of mRNA-loaded lipid nanoparticles for vaccine applications

Carolina Lopes et al.

Summary: This article highlights the importance of lipid nanoparticles in the field of RNA delivery, focusing on the use of microfluidic techniques in LNP preparation and the potential of spray drying technology in enhancing LNP stability.

EXPERT OPINION ON DRUG DELIVERY (2022)

Article Biochemistry & Molecular Biology

Lipid nanoparticle-encapsulated VEGFa siRNA facilitates cartilage formation by suppressing angiogenesis

Yi Chen et al.

Summary: Cartilage reconstruction is an urgent clinical demand, and inhibition of vascularization contributes to cartilage formation. In this study, a GelMA/Cells+VEGFa siRNA-LNPs scaffold was constructed to achieve superior cartilage regeneration by upregulating HIF-1α and FOXO transcription factors.

INTERNATIONAL JOURNAL OF BIOLOGICAL MACROMOLECULES (2022)

Review Infectious Diseases

Prevention of monkeypox with vaccines: a rapid review

Gregory A. Poland et al.

Summary: This review article provides a brief overview of human immune responses to orthopoxviruses, discusses the available vaccines to combat the monkeypox outbreak, and addresses concerns regarding the outbreak and the use of vaccines as a control measure. It emphasizes the clinical effectiveness of smallpox vaccines against monkeypox, supported by surveillance studies and animal experiments. The article also highlights the side-effects and limitations of smallpox vaccines and suggests the lessons learned from the COVID-19 pandemic should inform the collective response to this monkeypox outbreak and future outbreaks.

LANCET INFECTIOUS DISEASES (2022)

Article Multidisciplinary Sciences

Payload distribution and capacity of mRNA lipid nanoparticles

Sixuan Li et al.

Summary: The authors developed a method based on multi-laser cylindrical illumination confocal spectroscopy to quantitatively examine the mRNA and lipid contents in LNP formulations at the single-nanoparticle level. They revealed that a commonly referenced benchmark formulation containing DLin-MC3 as the ionizable lipid has mostly 2 mRNAs per loaded LNP and a presence of 40%-80% empty LNPs depending on the assembly conditions.

NATURE COMMUNICATIONS (2022)

Article Multidisciplinary Sciences

Endocytosis-like DNA uptake by cell wall-deficient bacteria

Renee Kapteijn et al.

Summary: A new endocytosis-like process has been discovered in cell wall-deficient bacteria, allowing them to engulf DNA and other extracellular materials. This process differs from the traditional mechanisms of horizontal gene transfer and requires energy. The findings suggest a possible mechanism for early life forms to acquire essential resources before the development of the bacterial cell wall.

NATURE COMMUNICATIONS (2022)

Article Pharmacology & Pharmacy

Development of a Combined Lipid-Based Nanoparticle Formulation for Enhanced siRNA Delivery to Vascular Endothelial Cells

Yutong He et al.

Summary: In this study, a lipid-based nanoparticle (LNP) formulation consisting of permanently charged cationic lipid-DOTAP and conditionally ionized cationic lipid-MC3 (DOTAP/MC3) was developed for enhanced siRNA delivery into endothelial cells (EC). The DOTAP/MC3 LNP formulation showed the best transfection efficiency compared to single DOTAP or MC3-based LNPs both in primary EC in vitro and in endothelium in zebrafish. The high transfection activity of the DOTAP/MC3 LNP formulation was achieved by improved endothelial cell association mediated by DOTAP and efficient siRNA intracellular release triggered by MC3.

PHARMACEUTICS (2022)

Review Biotechnology & Applied Microbiology

Lipid-mRNA nanoparticles landscape for cancer therapy

Yin Li et al.

Summary: The intracellular delivery of mRNA technique has brought hope for COVID-19 vaccines and cancer treatment. However, efficient and non-toxic delivery of therapeutic mRNAs remains a challenge. Lipid nanoparticles have emerged as powerful tools for mRNA delivery in cancer therapy.

FRONTIERS IN BIOENGINEERING AND BIOTECHNOLOGY (2022)

Article Virology

LNP-CpG ODN-adjuvanted varicella-zoster virus glycoprotein E induced comparable levels of immunity with ShingrixTM in VZV-primed mice

Ning Luan et al.

Summary: The LNP-CpG-adjuvanted VZV-gE vaccine developed in this study demonstrated comparable immunogenicity and cell-mediated immunity levels to ShingrixTM in a mouse model, suggesting its potential as a more cost-effective zoster vaccine.

VIROLOGICA SINICA (2022)

Article Virology

A multiple-target mRNA-LNP vaccine induces protective immunity against experimental multi-serotype DENV in mice

Lihong He et al.

Summary: The study developed a modified mRNA vaccine consisting of envelope domain III (E-DIII) and non-structural protein 1 (NS1) coated with lipid nanoparticles for dengue virus (DENV). The vaccine showed the ability to block all four types of DENV infection in vitro and induce a robust antiviral immune response. In a mouse model, the vaccine limited DENV transmission and eliminated vascular leakage.

VIROLOGICA SINICA (2022)

Review Engineering, Biomedical

Lipid Nanoparticles for Drug Delivery

Letao Xu et al.

Summary: This article provides an overview of lipid nanoparticles, including their different types, preparation methods, and applications in drug delivery. It also summarizes the challenges and potential future studies for lipid nanoparticles to achieve wider clinical and pharmaceutical applications.

ADVANCED NANOBIOMED RESEARCH (2022)

Review Materials Science, Biomaterials

Approaches towards biomaterial-mediated gene editing for cancer immunotherapy

Sydney R. Shannon et al.

Summary: Gene therapies are revolutionizing the treatment of many human diseases, and it is important to consider both the delivery materials and cargos for maximum efficacy and safety. Non-viral delivery mechanisms and CRISPR technology have great potential in immune cell gene editing, but there are challenges that need to be overcome.

BIOMATERIALS SCIENCE (2022)

Review Biochemistry & Molecular Biology

Advances in COVID-19 mRNA vaccine development

Enyue Fang et al.

Summary: This review summarizes the current knowledge on COVID-19 mRNA vaccines, including their structural characteristics, antigen design strategies, delivery systems, industrialization potential, quality control, latest clinical trials, real-world data, and the future directions in the development of preventive mRNA vaccines for major infectious diseases.

SIGNAL TRANSDUCTION AND TARGETED THERAPY (2022)

Review Biochemistry & Molecular Biology

Self-amplifying RNA vaccines for infectious diseases

Kristie Bloom et al.

Summary: Vaccinology is moving towards synthetic RNA platforms, which offer rapid, scalable, and cell-free manufacturing of prophylactic and therapeutic vaccines. Self-amplifying RNAs have shown enhanced antigen expression at lower doses compared to conventional mRNA, potentially improving immunity and becoming important vaccine candidates.

GENE THERAPY (2021)

Article Biology

Ionization and structural properties of mRNA lipid nanoparticles influence expression in intramuscular and intravascular administration

Manuel J. Carrasco et al.

Summary: The study reports a method that can predict the pKa of lipid nanoparticles (LNPs) from the structure of the ionizable lipid, and investigates the delivery efficiency and factors influencing LNPs both in vitro and in vivo. It was also found that LNPs with more negative charge lead to higher off-target systemic mRNA expression.

COMMUNICATIONS BIOLOGY (2021)

Article Engineering, Biomedical

mRNA Delivery Using Bioreducible Lipidoid Nanoparticles Facilitates Neural Differentiation of Human Mesenchymal Stem Cells

Xuewei Zhao et al.

Summary: The study developed a new method using lipidoid nanoparticles to deliver mRNA into human mesenchymal stem cells, improved delivery efficacy by adding cholesterol and other excipients, and successfully achieved CRISPR-based gene knockdown to facilitate MSCs transdifferentiation into neural-like lineage.

ADVANCED HEALTHCARE MATERIALS (2021)

Review Chemistry, Multidisciplinary

Developing Biodegradable Lipid Nanoparticles for Intracellular mRNA Delivery and Genome Editing

Min Qiu et al.

Summary: mRNA technology has garnered significant attention for its potential in revolutionizing medicine, particularly in areas such as vaccination and cancer immunotherapy. Advances in synthetic lipid nanoparticles (LNPs) as delivery systems have shown promise in facilitating mRNA-based therapies.

ACCOUNTS OF CHEMICAL RESEARCH (2021)

Article Chemistry, Multidisciplinary

Polymersomes as Stable Nanocarriers for a Highly Immunogenic and Durable SARS-CoV-2 Spike Protein Subunit Vaccine

Jian Hang Lam et al.

Summary: This study describes a subunit vaccine based on the SARS-CoV-2 spike protein coadministered with CpG adjuvant, encapsulated with a proprietary artificial cell membrane (ACM) polymersome technology to enhance immunogenicity. In mice experiments, two doses of this formulation elicited robust neutralizing antibody titers that persisted for at least 40 days and produced functional memory CD4(+) and CD8(+) T cells.

ACS NANO (2021)

Article Microbiology

D614G Spike Mutation Increases SARS CoV-2 Susceptibility to Neutralization

Drew Weissman et al.

Summary: The D614G mutation in the spike protein of SARS-CoV-2 is not expected to hinder current vaccine development, as viruses with the G614 spike are more susceptible to neutralization, indicating that the mutation may not affect vaccine efficacy.

CELL HOST & MICROBE (2021)

Article Medicine, Research & Experimental

Engineering of the current nucleoside-modified mRNA-LNP vaccines against SARS-CoV-2

Javier T. Granados-Riveron et al.

Summary: The review highlights the rapid development of mRNA-based COVID-19 vaccines within less than a year after the first SARS-CoV-2 infection. These vaccines elicit broadly protective immune responses and are amenable to rapid and flexible manufacturing processes, aiding in an agile response to the current pandemic.

BIOMEDICINE & PHARMACOTHERAPY (2021)

Article Pharmacology & Pharmacy

Lumasiran: First Approval

Lesley J. Scott et al.

Summary: Lumasiran is a drug developed for the treatment of primary hyperoxaluria type 1 by targeting a specific gene to inhibit the synthesis of toxic metabolites. It received approvals in the EU and USA, marking its global recognition.

DRUGS (2021)

Review Chemistry, Physical

Opportunities for innovation: Building on the success of lipid nanoparticle vaccines

Jessica Huang et al.

Summary: Lipid nanoparticle (LNP) formulations of messenger RNA (mRNA) have shown high efficacy as vaccines against SARS-CoV-2, highlighting their potential as a delivery system for next generation biological therapies. Key considerations for engineering LNPs as a vaccine delivery system include improving mRNA protection, optimizing cytosolic delivery, targeting specific cells, minimizing side effects, and controlling RNA release. Innovations in LNP vaccines are likely to accelerate with recent successes, with implications for a wide range of therapeutic applications beyond vaccination such as gene therapy.

CURRENT OPINION IN COLLOID & INTERFACE SCIENCE (2021)

Article Medicine, Research & Experimental

Enhanced Delivery of siRNA to Retinal Ganglion Cells by Intravitreal Lipid Nanoparticles of Positive Charge

Xiaonan Huang et al.

Summary: Positively charged LNPs have demonstrated specific gene downregulation in the RGC layer, showing potential for the treatment of RGC-associated retinal diseases.

MOLECULAR PHARMACEUTICS (2021)

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)

Article Multidisciplinary Sciences

A noninflammatory mRNA vaccine for treatment of experimental autoimmune encephalomyelitis

Christina Krienke et al.

Summary: The study demonstrates the potential use of modified m1 Psi mRNA in the treatment of multiple sclerosis, showing its ability to suppress the disease, promote the development of T-reg cell populations, and enhance bystander immunosuppression.

SCIENCE (2021)

Article Immunology

Lipid nanoparticles enhance the efficacy of mRNA and protein subunit vaccines by inducing robust T follicular helper cell and humoral responses

Mohamad-Gabriel Alameh et al.

Summary: Lipid nanoparticle (LNP)-encapsulated nucleoside-modified mRNA vaccines have intrinsic adjuvant activity that enhances durable and protective antibody responses. Compared to the widely used MF59-like adjuvant AddaVax, this LNP formulation outperforms and relies on specific components and cytokine induction for its adjuvant activity.

IMMUNITY (2021)

Article Immunology

Proof-of-concept of a low-dose unmodified mRNA-based rabies vaccine formulated with lipid nanoparticles in human volunteers: A phase 1 trial

Cassandra Aldrich et al.

Summary: Immune responses to rabies virus glycoprotein (RABV-G)-mRNA vaccine were dependent on the route of administration, necessitating specialized devices. The results showed that the 1 or 2 microgram doses were well tolerated and elicited rabies neutralizing antibody responses that met WHO criteria in all recipients.

VACCINE (2021)

Article Pharmacology & Pharmacy

Design and Characterization of Elastic Artificial Skin Containing Adenosine-Loaded Solid Lipid Nanoparticles for Treating Wrinkles

Sooho Yeo et al.

Summary: In this study, an elastic artificial skin for treating wrinkles with poor skin permeation of adenosine was developed, along with a lipid-based nano-carrier to enhance the skin permeation of adenosine. The results showed that the elastic artificial skin incorporated with adenosine-loaded solid lipid nanoparticles exhibited better drug release characteristics.

PHARMACEUTICS (2021)

Review Pharmacology & Pharmacy

Self-assembled mRNA vaccines

Jeonghwan Kim et al.

Summary: mRNA vaccines, emerging as front-runners in the COVID-19 vaccine race, utilize advancements in RNA technology, vaccinology, and nanotechnology to design self-assembled nanoparticles for vaccine delivery, showing tremendous promise and readiness for clinical use.

ADVANCED DRUG DELIVERY REVIEWS (2021)

Article Chemistry, Multidisciplinary

Zwitterionic Phospholipidation of Cationic Polymers Facilitates Systemic mRNA Delivery to Spleen and Lymph Nodes

Shuai Liu et al.

Summary: The study introduces a rational design and combinatorial synthesis of zwitterionic phospholipidated polymers (ZPPs) for efficient mRNA delivery to spleen and lymph nodes, with potential for substantial immunotherapeutic applications.

JOURNAL OF THE AMERICAN CHEMICAL SOCIETY (2021)

Review Biochemistry & Molecular Biology

Cationic lipids for gene delivery: many players, one goal

Federica Ponti et al.

Summary: Lipid-based carriers are widely used as non-viral vectors for gene expression and gene silencing, due to their ease of synthesis and modification. While significant progress has been made in developing cationic lipids, further research is needed for more effective lipid vectors for gene transfection. This review emphasizes the importance of designing each constituent domain of cationic lipids and how it affects their performance.

CHEMISTRY AND PHYSICS OF LIPIDS (2021)

Article Biochemistry & Molecular Biology

A phase 1 trial of lipid-encapsulated mRNA encoding a monoclonal antibody with neutralizing activity against Chikungunya virus

Allison August et al.

Summary: The study evaluated the safety and pharmacology of mRNA-1944 as a treatment for Chikungunya virus (CHIKV) infection, showing good safety and tolerability in healthy participants. Additionally, mRNA-1944 demonstrated the ability to produce effective neutralizing antibodies, suggesting potential for treating CHIKV infection.

NATURE MEDICINE (2021)

Article Chemistry, Multidisciplinary

Lipid nanoparticles loaded with ribonucleoprotein-oligonucleotide complexes synthesized using a microfluidic device exhibit robust genome editing and hepatitis B virus inhibition

Yuichi Suzuki et al.

Summary: This study presents a lipid nanoparticle (LNP)-based CRISPR/Cas ribonucleoprotein (RNP) delivery nanoplatform synthesized using a clinically relevant mixer-equipped microfluidic device, which avoids DNA cleavage activity and Cas enzyme aggregation. The optimized formulation, identified through 2 steps of design of experiments, showed excellent gene disruption and base substitution efficiencies without apparent cytotoxicity. The addition of negative charges to the RNPs by complexing single-stranded oligonucleotide (ssON) significantly enhanced the delivery of both Cas9 and Cpf1 RNPs.

JOURNAL OF CONTROLLED RELEASE (2021)

Review Biochemistry & Molecular Biology

mRNA vaccine for cancer immunotherapy

Lei Miao et al.

Summary: mRNA vaccines have emerged as a promising platform for cancer immunotherapy, showcasing high potency, safe administration, rapid development potentials, and cost-effective manufacturing. However, challenges such as stability, innate immunogenicity, and inefficient in vivo delivery restrict their applications. In addressing these challenges, structural modifications and formulation methods are being researched to enhance the anti-tumor immune response.

MOLECULAR CANCER (2021)

Article Multidisciplinary Sciences

On the mechanism of tissue-specific mRNA delivery by selective organ targeting nanoparticles

Sean A. Dilliard et al.

Summary: SORT nanoparticles have been developed to deliver genetic medicines to non-liver tissues by incorporating a supplemental SORT molecule and recruiting specific proteins to the nanoparticle surface, bypassing the liver accumulation barrier typically seen with traditional LNPs.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2021)

Article Multidisciplinary Sciences

Lipid nanoparticle-mediated codelivery of Cas9 mRNA and single-guide RNA achieves liver-specific in vivo genome editing of Angptl3

Min Qiu et al.

Summary: Loss-of-function mutations in Angiopoietin-like 3 (Angptl3) are linked to reduced blood lipid levels, making it a potential therapeutic target for lipid metabolism disorders. A lipid nanoparticle delivery system successfully edited Angptl3 gene in vivo, leading to significant reductions in lipid levels without off-target effects or toxicity, highlighting the potential of this platform for safe and effective gene editing therapies.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2021)

Article Multidisciplinary Sciences

Spatiotemporally programmable cascade hybridization of hairpin DNA in polymeric nanoframework for precise siRNA delivery

Feng Li et al.

Summary: DNA nanostructures have been used as carriers for gene delivery, but spatiotemporally programmable assembly of DNA under nanoconfinement is challenging due to DNA complexity. A strategy using cascade hybridization chain reaction of DNA hairpins in polymeric nanoframework enables precise siRNA delivery. The nanoframework provides stability and ease of functionalization, addressing DNA complexity issues. Phenylboronate installation on nanoframework enhances cellular uptake and lysosomal escape.

NATURE COMMUNICATIONS (2021)

Article Biotechnology & Applied Microbiology

Novel fusion peptide-mediated siRNA delivery using self-assembled nanocomplex

Yeong Chae Ryu et al.

Summary: Novel synergy fusion peptides SPACE and oligoarginine improved cellular uptake and gene silencing effect of siRNA in vitro, as well as retention and gene silencing effects of siRNAs in vivo. The nanocomplexes using fusion peptides showed enhanced cellular uptake and gene silencing effects via lipid raft-mediated endocytosis pathway, making them a potential safe and efficient platform for delivering siRNAs for gene silencing therapies.

JOURNAL OF NANOBIOTECHNOLOGY (2021)

Article Medicine, Research & Experimental

Functionalized lipid nanoparticles for subcutaneous administration of mRNA to achieve systemic exposures of a therapeutic protein

Nigel Davies et al.

Summary: Subcutaneous administration of mRNA formulated in LNPs can achieve measurable plasma exposure of secreted proteins, but is associated with dose-limiting inflammatory responses. Incorporating aliphatic ester prodrugs of anti-inflammatory steroids within LNPs can suppress inflammation and prolong protein expression, making it a viable approach for achieving systemic levels of therapeutic proteins.

MOLECULAR THERAPY-NUCLEIC ACIDS (2021)

Article Multidisciplinary Sciences

An ionizable lipid toolbox for RNA delivery

Xuexiang Han et al.

Summary: In recent years, RNA therapeutics has seen significant growth, largely due to decades of research on lipid nanoparticles, particularly its key component - ionizable lipid. This commentary discusses the major types of ionizable lipids and offers perspectives for future development.

NATURE COMMUNICATIONS (2021)

Article Multidisciplinary Sciences

Cytosolic protein delivery using pH-responsive, charge-reversible lipid nanoparticles

Yusuke Hirai et al.

Summary: This study evaluated the applicability of DOP-DEDA-based LNPs for intracellular protein delivery, successfully encapsulating a negatively charged GFP analog and facilitating cellular uptake and endosomal release.

SCIENTIFIC REPORTS (2021)

Review Biotechnology & Applied Microbiology

Cytosolic delivery of nucleic acids: The case of ionizable lipid nanoparticles

Michele Schlich et al.

Summary: Ionizable lipid nanoparticles (LNPs) are the most advanced nano-delivery system for therapeutic nucleic acids, with the development of ionizable lipids playing a key role in their success. Overcoming challenges in RNA delivery, such as endosomal escape, is crucial for ensuring the therapeutic activity of RNA molecules. Understanding the mechanisms of endosomal escape and improving cytosolic delivery are important for developing future LNPs with enhanced nucleic acid delivery capabilities.

BIOENGINEERING & TRANSLATIONAL MEDICINE (2021)

Article Chemistry, Multidisciplinary

mRNA Delivery of a Bispecific Single-Domain Antibody to Polarize Tumor-Associated Macrophages and Synergize Immunotherapy against Liver Malignancies

Ying Wang et al.

Summary: Liver malignancies are resistant to immune checkpoint inhibition therapy due to the presence of tumor-associated macrophages (TAMs) that induce immunosuppression. The study identified CCL2 and CCL5 as major chemokines responsible for TAM infiltration in liver malignancies and developed a bispecific antibody BisCCL2/5i to reverse this immunosuppressive process.

ADVANCED MATERIALS (2021)

Article Biochemistry & Molecular Biology

Secondary Structure-Driven Self-Assembly of Thiol-Reactive Polypept(o)ides

Tobias A. Bauer et al.

Summary: The formation of secondary structures in polypeptides influences their self-assembly behavior, with copolymers containing antiparallel beta-sheets showing a strong tendency to form worm-like micelles. Different secondary structures lead to distinct morphologies in the resulting nanostructures, highlighting the potential of secondary structure-driven self-assembly in synthetic polypeptides.

BIOMACROMOLECULES (2021)

Article Biochemical Research Methods

Recent developments in microfluidic synthesis of artificial cell-like polymersomes and liposomes for functional bioreactors

Hanjin Seo et al.

Summary: Recent advances in droplet microfluidics have enabled the fabrication of versatile vesicles mimicking cellular membranes, which effectively enclose an aqueous core for various biological reactions. These artificial cell-like vesicles have shown potential in synthetic biology and biomedical applications such as drug delivery and biosensors.

BIOMICROFLUIDICS (2021)

Review Pharmacology & Pharmacy

mRNA-lipid nanoparticle COVID-19 vaccines: Structure and stability

Linde Schoenmaker et al.

Summary: The current mRNA-lipid nanoparticle COVID-19 vaccines require storage at low temperatures due to instability. Improving mRNA nucleotide composition, understanding the impact of mRNA environment within LNPs, and exploring drying techniques can enhance vaccine stability.

INTERNATIONAL JOURNAL OF PHARMACEUTICS (2021)

Article Immunology

Development of multivalent mRNA vaccine candidates for seasonal or pandemic influenza

Sudha Chivukula et al.

Summary: The recent approval of mRNA vaccines for emergency use against COVID-19 is likely to accelerate the development of mRNA-based vaccines targeting a wide range of infectious diseases. This vaccine modality promises comparable potency while accelerating the development and deployment of vaccine doses. Demonstrated successfully for single antigen vaccines, the technology could be optimized for complex multi-antigen vaccines.

NPJ VACCINES (2021)

Article Biotechnology & Applied Microbiology

3D Printed Integrated Multi-Layer Microfluidic Chips for Ultra-High Volumetric Throughput Nanoliposome Preparation

Han Shan et al.

Summary: This study presents a method for ultra-high volumetric throughput nanoliposome preparation using 3D printed microfluidic chips. By adopting a three-layer layout for microchannels, the microfluidic chip achieved a total flow rate up to 474 ml min(-1) and controlled the liposome size to be as small as 80 nm. The experimental results demonstrate the great potential of the 3D printed integrated microfluidic chip in targeting drug delivery systems.

FRONTIERS IN BIOENGINEERING AND BIOTECHNOLOGY (2021)

Article Engineering, Biomedical

Study the lipidoid nanoparticle mediated genome editing protein delivery using 3D intestinal tissue model

Tao Yang et al.

Summary: The study demonstrates that synthetic lipid nanoparticles and protein cargo nanocomplexes can successfully cross the intestinal epithelial layer and deliver the protein cargo to targeted cells. These nanocomplexes can penetrate intestinal monolayer cells by temporarily interrupting tight junctions between cells, inducing gene recombination inside the cells below.

BIOACTIVE MATERIALS (2021)

Article Medicine, General & Internal

Anticancer Drugs Approved by the US Food and Drug Administration From 2009 to 2020 According to Their Mechanism of Action

Timothee Olivier et al.

Summary: This study found that between 2009 and 2020, the majority of all approved anticancer drugs were based on next-in-class indications, with only a minority being based on new mechanisms of action. The study suggests further consideration of incentives for drug development to prioritize novel and transformative anticancer drugs.

JAMA NETWORK OPEN (2021)

Review Biochemistry & Molecular Biology

mRNA therapeutics in cancer immunotherapy

Jan D. Beck et al.

Summary: Synthetic mRNA serves as a versatile template for protein synthesis and has a wide range of pharmaceutical applications, including cancer immunotherapy. Strategies such as stimulating pattern recognition receptors and nucleoside modification enhance the effectiveness and safety of mRNA vaccines.

MOLECULAR CANCER (2021)

Article Biotechnology & Applied Microbiology

Lipid-nanoparticle-encapsulated mRNA vaccines induce protective memory CD8 T cells against a lethal viral infection

Cory J. Knudson et al.

Summary: Different doses of modified mRNA vaccines can induce strong CD8 T cell responses, providing protective immunity and increasing the memory CD8 T cell pool, which can be used to create CD8 T cell-based vaccines against viral infections.

MOLECULAR THERAPY (2021)

Review Immunology

Neoantigen: A New Breakthrough in Tumor Immunotherapy

Zheying Zhang et al.

Summary: Cancer immunotherapy enhances the body's immune response to target and eliminate cancer cells, with a focus on the emerging approach of tumor neoantigens. Studies have shown the relationship between neoantigens and T cell recognition of cancer cells, highlighting the potential of vaccines developed against these specific antigens in clinical trials.

FRONTIERS IN IMMUNOLOGY (2021)

Article Multidisciplinary Sciences

Scaffold-mediated CRISPR-Cas9 delivery system for acute myeloid leukemia therapy

Tzu-Chieh Ho et al.

Summary: Utilizing a nano-based delivery system targeting leukemia stem cells in the bone marrow microenvironment shows promise in attenuating LSC growth and improving AML therapy. Precise gene editing through IL1RAP knockout results in reduced colony-forming capacity, leading to decreased leukemic burden.

SCIENCE ADVANCES (2021)

Article Immunology

Lipid nanoparticle encapsulated nucleoside-modified mRNA vaccines elicit polyfunctional HIV-1 antibodies comparable to proteins in nonhuman primates

Kevin O. Saunders et al.

Summary: mRNA-LNP vaccines show promising immunogenicity in inducing polyfunctional antibodies against HIV-1 and Zika virus, with even low doses being effective in macaques. Such vaccines may offer superior or comparable immunogenicity compared to adjuvanted recombinant protein vaccines in primates.

NPJ VACCINES (2021)

Review Immunology

Lipid-Based Nanoparticles in the Clinic and Clinical Trials: From Cancer Nanomedicine to COVID-19 Vaccines

Thai Thanh Hoang Thi et al.

Summary: This review highlights the importance of lipid-based nanoparticles in the development of COVID-19 vaccines and other nanomedicines, focusing on innovations that have obtained regulatory approval or are in clinical trials. It also discusses the physicochemical properties required for specific applications, emphasizes the differences in requirements for delivering different cargos, and introduces current challenges that need further development.

VACCINES (2021)

Review Chemistry, Multidisciplinary

Lipid Nanoparticles-From Liposomes to mRNA Vaccine Delivery, a Landscape of Research Diversity and Advancement

Rumiana Tenchov et al.

Summary: Lipid nanoparticles (LNPs) have become promising vehicles in the pharmaceutical industry for delivering various therapeutics, particularly highlighted in the COVID-19 mRNA vaccines. LNPs exhibit versatility and physical stability, allowing for targeted delivery of therapeutic drugs to specific locations within the body. Liposomes, an early version of LNPs, have already been widely applied in medical practice.

ACS NANO (2021)

Review Engineering, Biomedical

From influenza to COVID-19: Lipid nanoparticle mRNA vaccines at the frontiers of infectious diseases

Emily H. Pilkington et al.

Summary: Vaccination is crucial in fighting infectious diseases, and mRNA vaccines formulated with lipid nanoparticles offer a faster and safer alternative for vaccination. Breakthroughs in mRNA-LNP vaccine development have shown promising results in combating infectious diseases and are currently being applied in clinical trials.

ACTA BIOMATERIALIA (2021)

Article Chemistry, Multidisciplinary

Construction of Messenger RNA (mRNA) Probes Delivered By Lipid Nanoparticles to Visualize Intracellular Protein Expression and Localization at Organelles

Weiyu Zhao et al.

Summary: The SOLAR and DOLAR mRNA probes are designed to encode fluorescent proteins with different organelle localization signals, allowing visualization of protein localizations at various organelles and investigation of protein trafficking from ribosomal machinery to specific organelles. The in vitro results show that SOLAR probes exhibit consistent organelle targeting capabilities, while DOLAR probes with different linkers display distinct targeting properties based on organelle localization signals. Additionally, these mRNA probes demonstrate organelle labeling ability in vivo when delivered by lipid nanoparticles, providing a unique tool to study cell organelles and potentially aid in the design of organelle-based therapies.

ADVANCED MATERIALS (2021)

Review Engineering, Biomedical

Microfluidic formulation of nanoparticles for biomedical applications

Sarah J. Shepherd et al.

Summary: Nanomedicine has advanced significantly in clinical applications, with microfluidic devices showing potential for generating reproducible nanoparticle formulations. These formulations enhance biomedical applications with controlled physical properties, pointing towards future production scale-independent formulations through parallelization and multi-step reactions.

BIOMATERIALS (2021)

Review Chemistry, Multidisciplinary

Lipids and Lipid Derivatives for RNA Delivery

Yuebao Zhang et al.

Summary: RNA-based therapeutics have shown great promise in treating diseases through various mechanisms, with lipid nanoparticles being the most widely used delivery system. The study of lipids, lipid derivatives, and lipid-derived macromolecules for RNA delivery has also made significant progress in recent years.

CHEMICAL REVIEWS (2021)

Article Pharmacology & Pharmacy

Antibody-siRNA conjugates (ARCs) using multifunctional peptide as a tumor enzyme cleavable linker mediated effective intracellular delivery of siRNA

Zhili Yu et al.

Summary: In this study, antibodies and siRNAs were successfully conjugated using a multifunctional peptide as a linker, allowing for the specific delivery of siRNAs to target tumor cells. The released siRNA entered targeted cells via cellular penetrating peptide, resulting in improved gene silencing efficiency. The designed ARCs achieved approximately 66.7% EGFP downregulation efficiency in nude mice xenografted with the HCT116-EGFP tumor model, demonstrating a prospective choice for safe and efficient siRNA delivery.

INTERNATIONAL JOURNAL OF PHARMACEUTICS (2021)

Article Chemistry, Multidisciplinary

Therapeutic inhibitory RNA in head and neck cancer via functional targeted lipid nanoparticles

Liyona Kampel et al.

Summary: The study demonstrated the therapeutic efficacy of antiE6/E7 siRNA delivered via targeted LNPs in a xenograft HPV-positive tumor model, showing superior suppression of HPV oncogenes and higher induction of apoptosis. Combining anti-EGFR antibodies with siRNA could enhance cargo delivery and mediate anti-tumor activity, successfully restricting tumor progression.

JOURNAL OF CONTROLLED RELEASE (2021)

Article Chemistry, Multidisciplinary

Scalable mRNA and siRNA Lipid Nanoparticle Production Using a Parallelized Microfluidic Device

Sarah J. Shepherd et al.

Summary: The study presents a scalable, parallelized microfluidic device (PMD) that can simultaneously operate 128 mixing channels, achieving over 100 times production rate compared to traditional methods for lipid nanoparticles (LNPs). Results demonstrate superior delivery of LNPS generated using PMD in mice, showing potential for emerging clinical applications such as RNA therapeutics and vaccines.

NANO LETTERS (2021)

Article Multidisciplinary Sciences

BNT162b2 vaccine induces neutralizing antibodies and poly-specific T cells in humans

Ugur Sahin et al.

Summary: The BNT162b2 vaccine shows 95% efficacy in preventing COVID-19 by boosting neutralizing antibody titres and activating specific T cell responses. The vaccine-induced immune response is broad and stable, lasting for a prolonged period, providing good coverage against various SARS-CoV-2 variants.

NATURE (2021)

Article Medicine, General & Internal

CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis

Julian D. Gillmore et al.

Summary: The gene-editing therapeutic agent NTLA-2001 effectively reduced serum TTR protein concentrations in patients with ATTR amyloidosis, showing potential safety and pharmacodynamic effects in clinical studies.

NEW ENGLAND JOURNAL OF MEDICINE (2021)

Article Multidisciplinary Sciences

Neoantigens elicit T cell responses in breast cancer

Takafumi Morisaki et al.

Summary: The study identified a positive correlation between neoantigen load and total number of mutations in breast cancer cells. In vitro coculture of neoantigen-peptide pulsed dendritic cells with lymphocytes successfully induced cytotoxic T lymphocytes. These results suggest the potential utility of neoantigen analysis in developing T cell response strategies.

SCIENTIFIC REPORTS (2021)

Article Virology

Future considerations for the mRNA-lipid nanoparticle vaccine platform

Botond Z. Igyarto et al.

Summary: mRNA-based LNPs are a promising new vaccine platform, but there are still many unanswered questions regarding their mechanism and potential side effects. Utilizing dendritic cell biology could help mitigate these risks.

CURRENT OPINION IN VIROLOGY (2021)

Article Medicine, Research & Experimental

Irf5 siRNA-loaded biodegradable lipid nanoparticles ameliorate concanavalin A-induced liver injury

Wataru Kawase et al.

Summary: RNA interference-based gene silencing drugs delivered by LNPs have shown potential for treating diseases, especially inflammatory disorders. LNPs efficiently deliver siRNA to macrophages, reducing inflammatory responses.

MOLECULAR THERAPY-NUCLEIC ACIDS (2021)

Article Immunology

A Neoantigen-Based Peptide Vaccine for Patients With Advanced Pancreatic Cancer Refractory to Standard Treatment

Zheling Chen et al.

Summary: This study enrolled 7 advanced pancreatic cancer patients and successfully applied neoantigen identification and selection, demonstrating that a personalized neoantigen-based peptide vaccine iNeo-Vac-P01 could improve the clinical efficacy limitations of pancreatic cancer.

FRONTIERS IN IMMUNOLOGY (2021)

Review Nanoscience & Nanotechnology

Lipid nanoparticles for mRNA delivery

Xucheng Hou et al.

Summary: Lipid nanoparticle-mRNA formulations have successfully entered clinical use as COVID-19 vaccines, representing a major milestone in mRNA therapeutics. This review discusses the design of lipid nanoparticles for mRNA delivery, emphasizing key points for clinical translation and preclinical studies of lipid nanoparticle-mRNA therapeutics for various diseases.

NATURE REVIEWS MATERIALS (2021)

Review Pharmacology & Pharmacy

In vivo delivery of CRISPR-Cas9 therapeutics: Progress and challenges

Matthew Behr et al.

Summary: CRISPR-Cas9-based genome editing has advanced to human clinical trials within less than a decade, but the low efficiency of in vivo delivery needs to be enhanced in order to fully realize its therapeutic potential.

ACTA PHARMACEUTICA SINICA B (2021)

Article Oncology

Impact of Neoantigen Expression and T-Cell Activation on Breast Cancer Survival

Wenjing Li et al.

Summary: Neoantigens are novel proteins derived from tumor-specific somatic mutations and have shown promise in breast cancer immunotherapy. High expression of neoantigens and activated T-cell status are associated with improved overall survival in breast cancer patients, indicating their potential as immunogenic agents for treatment.

CANCERS (2021)

Review Oncology

The Role of Neoantigens in Cancer Immunotherapy

Yueting Zhu et al.

Summary: Somatic mutation-derived neoantigens expressed on tumor cells can trigger anti-tumor T-cell responses with low immune tolerance. Technologies for identifying neoantigens based on whole-exome sequencing have shown promising therapeutic effects in personalized cancer vaccines and adoptive T cell therapies. Challenges in targeting neoantigens for cancer treatment are also discussed in this review.

FRONTIERS IN ONCOLOGY (2021)

Review Chemistry, Physical

Nanotechnology-Assisted RNA Delivery: From Nucleic Acid Therapeutics to COVID-19 Vaccines

Chiara Rinoldi et al.

Summary: This article reviews the latest developments in RNA therapy, focusing on the development, characteristics, and applications of nanotechnology-assisted RNA delivery drugs. It discusses the importance and challenges of RNA drugs in the field of treatment, as well as possible future directions for development.

SMALL METHODS (2021)

Review Engineering, Biomedical

Engineering Micro-Nanomaterials for Biomedical Translation

Yaping Chen et al.

Summary: Different types of engineered nano-bio interfaces, driven by various micro/nanoneedles, nanoparticles, organ-on-chip devices, and nanosubstrates, are advancing scientific and technological progress in biomedical research.

ADVANCED NANOBIOMED RESEARCH (2021)

Article Materials Science, Biomaterials

Boosting ionizable lipid nanoparticle-mediated in vivo mRNA delivery through optimization of lipid amine-head groups

Feng Ding et al.

Summary: The study demonstrates that the choice of amine-head group significantly influences the mRNA delivery efficacy in ionizable lipids, with spermine-based 114-iLNP showing great potential for mRNA therapeutics development.

BIOMATERIALS SCIENCE (2021)

Article Materials Science, Biomaterials

Identification of a potent ionizable lipid for efficient macrophage transfection and systemic anti-interleukin-1β siRNA delivery against acute liver failure

Feng Ding et al.

Summary: RNA interference therapy shows great potential in treating inflammatory diseases, but developing potent carrier materials for delivering siRNA to macrophages is challenging. This study designed ionizable lipid nanoparticles to carry siRNA for silencing IL-1 beta in macrophages, achieving efficient internalization and endosome escape. The optimized LNP/siIL-1 beta successfully silenced IL-1 beta in both activated cells and primary macrophages, indicating its potential for anti-inflammation therapies.

JOURNAL OF MATERIALS CHEMISTRY B (2021)

Review Oncology

The Changing Landscape of Therapeutic Cancer Vaccines-Novel Platforms and Neoantigen Identification

Jessica Jou et al.

Summary: Therapeutic cancer vaccines aim to create and enhance tumor-specific T-cell responses, but face challenges in achieving success. Researchers are exploring various strategies to overcome immunosuppressive mechanisms in the tumor microenvironment, improve vaccine delivery platforms, and utilize combination therapies.

CLINICAL CANCER RESEARCH (2021)

Review Materials Science, Multidisciplinary

Flash technology-based self-assembly in nanoformulation: Fabrication to biomedical applications

Hanze Hu et al.

Summary: Advances in nanoformulation using flash-based technology have led to the development of nanoscale tools for biosensing, imaging, and drug delivery in biomedicine. This review discusses the theoretical and experimental principles, mixing device design, and applications of flash-based self-assembly in biomedicine, focusing particularly on flash nanocomplexation (FNC) and flash nanoprecipitation (FNP). Challenges and future directions for flash-based nanoformulation in biomedicine are also highlighted.

MATERIALS TODAY (2021)

Review Pharmacology & Pharmacy

Nanoplatforms for mRNA Therapeutics

Chaoyang Meng et al.

Summary: mRNA-based therapeutics are a unique class of drugs for treating human diseases, which require packaging into specially designed delivery platforms to exert their effects. Nanotechnology-based delivery platforms have been developed for effective mRNA delivery, with potential applications in biotechnology and clinical settings.

ADVANCED THERAPEUTICS (2021)

Article Pharmacology & Pharmacy

Freeze-drying cycle optimization for the rapid preservation of protein-loaded liposomal formulations

Maryam T. Hussain et al.

INTERNATIONAL JOURNAL OF PHARMACEUTICS (2020)

Review Biotechnology & Applied Microbiology

The challenge and prospect of mRNA therapeutics landscape

Yuhua Weng et al.

BIOTECHNOLOGY ADVANCES (2020)

Article Engineering, Chemical

Performance Evaluation of Liquid Mixing in a T-Junction Passive Micromixer with a Twisted Tape Insert

Jundika C. Kurnia et al.

INDUSTRIAL & ENGINEERING CHEMISTRY RESEARCH (2020)

Article Multidisciplinary Sciences

Cryo-EM structure of the 2019-nCoV spike in the prefusion conformation

Daniel Wrapp et al.

SCIENCE (2020)

Article Multidisciplinary Sciences

Nucleotide lipid-based hydrogel as a new biomaterial ink for biofabrication

Berangere Dessane et al.

SCIENTIFIC REPORTS (2020)

Article Pharmacology & Pharmacy

Using microfluidics for scalable manufacturing of nanomedicines from bench to GMP: A case study using protein-loaded liposomes

Cameron Webb et al.

INTERNATIONAL JOURNAL OF PHARMACEUTICS (2020)

Article Pharmacology & Pharmacy

Charge-reversible lipid derivative: A novel type of pH-responsive lipid for nanoparticle-mediated siRNA delivery

Yusuke Hirai et al.

INTERNATIONAL JOURNAL OF PHARMACEUTICS (2020)

Article Multidisciplinary Sciences

Synergistic lipid compositions for albumin receptor mediated delivery of mRNA to the liver

Lei Miao et al.

NATURE COMMUNICATIONS (2020)

Article Multidisciplinary Sciences

Systemic modified messenger RNA for replacement therapy in alpha 1-antitrypsin deficiency

Ahmad Karadagi et al.

SCIENTIFIC REPORTS (2020)

Article Chemistry, Multidisciplinary

Mechanical Properties Determination of DMPC, DPPC, DSPC, and HSPC Solid-Ordered Bilayers

Dominik Drabik et al.

LANGMUIR (2020)

Article Biotechnology & Applied Microbiology

A Multi-Targeting, Nucleoside-Modified mRNA Influenza Virus Vaccine Provides Broad Protection in Mice

Alec W. Freyn et al.

MOLECULAR THERAPY (2020)

Article Multidisciplinary Sciences

Targeted gene silencing in vivo by platelet membrane-coated metal-organic framework nanoparticles

Jia Zhuang et al.

SCIENCE ADVANCES (2020)

Article Chemistry, Multidisciplinary

Deconvoluting Lipid Nanoparticle Structure for Messenger RNA Delivery

Yulia Eygeris et al.

NANO LETTERS (2020)

Article Nanoscience & Nanotechnology

Selective organ targeting (SORT) nanoparticles for tissue-specific mRNA delivery and CRISPR-Cas gene editing

Qiang Cheng et al.

NATURE NANOTECHNOLOGY (2020)

Article Medicine, General & Internal

Two Phase 3 Trials of Inclisiran in Patients with Elevated LDL Cholesterol

Kausik K. Ray et al.

NEW ENGLAND JOURNAL OF MEDICINE (2020)

Article Multidisciplinary Sciences

Impact of mRNA chemistry and manufacturing process on innate immune activation

Jennifer Nelson et al.

SCIENCE ADVANCES (2020)

Article Multidisciplinary Sciences

Development of a potent Zika virus vaccine using self-amplifying messenger RNA

Kate Luisi et al.

SCIENCE ADVANCES (2020)

Article Chemistry, Physical

Surface pKa of Saturated Carboxylic Acids at the Air/Water Interface: A Quantum Chemical Approach

Yuri B. Vysotsky et al.

JOURNAL OF PHYSICAL CHEMISTRY C (2020)

Article Multidisciplinary Sciences

An RNA vaccine drives immunity in checkpoint-inhibitor-treated melanoma

Ugur Sahin et al.

NATURE (2020)

Article Medicine, General & Internal

An mRNA Vaccine against SARS-CoV-2-Preliminary Report

L. A. Jackson et al.

NEW ENGLAND JOURNAL OF MEDICINE (2020)

Article Oncology

Intratumoral IL12 mRNA Therapy Promotes TH1 Transformation of the Tumor Microenvironment

Susannah L. Hewitt et al.

CLINICAL CANCER RESEARCH (2020)

Article Chemistry, Multidisciplinary

Manipulating the function of tumor-associated macrophages by siRNA-loaded lipid nanoparticles for cancer immunotherapy

Nour Shobaki et al.

JOURNAL OF CONTROLLED RELEASE (2020)

Article Medicine, General & Internal

Safety and Immunogenicity of Two RNA-Based Covid-19 Vaccine Candidates

Edward E. Walsh et al.

NEW ENGLAND JOURNAL OF MEDICINE (2020)

Article Medicine, General & Internal

Safety and Immunogenicity of SARS-CoV-2 mRNA-1273 Vaccine in Older Adults

E. J. Anderson et al.

NEW ENGLAND JOURNAL OF MEDICINE (2020)

Review Chemistry, Multidisciplinary

Advances in Lipid Nanoparticles for mRNA-Based Cancer Immunotherapy

Maria L. Guevara et al.

FRONTIERS IN CHEMISTRY (2020)

Article Multidisciplinary Sciences

Functionalized lipid-like nanoparticles for in vivo mRNA delivery and base editing

Xinfu Zhang et al.

SCIENCE ADVANCES (2020)

Review Engineering, Biomedical

Studies of nanoparticle delivery with in vitro bio-engineered microtissues

Mingze Sun et al.

BIOACTIVE MATERIALS (2020)

Article Nanoscience & Nanotechnology

Polysarcosine-Functionalized Lipid Nanoparticles for Therapeutic mRNA Delivery

Sara S. Nogueira et al.

ACS APPLIED NANO MATERIALS (2020)

Article Multidisciplinary Sciences

The effects of PEGylation on LNP based mRNA delivery to the eye

Renee C. Ryals et al.

PLOS ONE (2020)

Article Chemistry, Multidisciplinary

The use of design of experiments with multiple responses to determine optimal formulations for in vivo hepatic mRNA delivery

Akari Hashiba et al.

JOURNAL OF CONTROLLED RELEASE (2020)

Review Chemistry, Multidisciplinary

In vivoandin vitrostudies of antisense oligonucleotides - a review

Anna Kilanowska et al.

RSC ADVANCES (2020)

Review Cell & Tissue Engineering

Messenger RNA Delivery for Tissue Engineering and Regenerative Medicine Applications

Siddharth Patel et al.

TISSUE ENGINEERING PART A (2019)

Article Chemistry, Multidisciplinary

Rigorous control of vesicle-forming lipid pKa by fluorine-conjugated bioisosteres for gene-silencing with siRNA

Ayaka Okamoto et al.

JOURNAL OF CONTROLLED RELEASE (2019)

Review Biotechnology & Applied Microbiology

Delivering the Messenger: Advances in Technologies for Therapeutic mRNA Delivery

Piotr S. Kowalski et al.

MOLECULAR THERAPY (2019)

Review Biotechnology & Applied Microbiology

Recent Developments in mRNA-Based Protein Supplementation Therapy to Target Lung Diseases

Itishri Sahu et al.

MOLECULAR THERAPY (2019)

Article Chemistry, Analytical

Optimization of micromixer with triangular baffles for chemical process in millidevices

Harrson S. Santana et al.

SENSORS AND ACTUATORS B-CHEMICAL (2019)

Article Medicine, Research & Experimental

Characterization of HIV-1 Nucleoside-Modified mRNA Vaccines in Rabbits and Rhesus Macaques

Norbert Pardi et al.

MOLECULAR THERAPY-NUCLEIC ACIDS (2019)

Article Materials Science, Multidisciplinary

High Throughput Nanoliposome Formation Using 3D Printed Microfluidic Flow Focusing Chips

Zhu Chen et al.

ADVANCED MATERIALS TECHNOLOGIES (2019)

Article Medicine, Research & Experimental

Optimization of Lipid Nanoparticles for Intramuscular Administration of mRNA Vaccines

Kimberly J. Hassett et al.

MOLECULAR THERAPY-NUCLEIC ACIDS (2019)

Article Nanoscience & Nanotechnology

Surface Coating Approach to Overcome Mucosal Entrapment of DNA Nanoparticles for Oral Gene Delivery of Glucagon-like Peptide 1

Tianqi Nie et al.

ACS APPLIED MATERIALS & INTERFACES (2019)

Article Chemistry, Multidisciplinary

Lipid nanoparticles for delivery of messenger RNA to the back of the eye

Siddharth Patel et al.

JOURNAL OF CONTROLLED RELEASE (2019)

Article Chemistry, Multidisciplinary

Multicistronic IVT mRNA for simultaneous expression of multiple fluorescent proteins

Kyuri Lee et al.

JOURNAL OF INDUSTRIAL AND ENGINEERING CHEMISTRY (2019)

Article Chemistry, Medicinal

Mannosylation of LNP Results in Improved Potency for Self-Amplifying RNA (SAM) Vaccines

Roshan Goswami et al.

ACS INFECTIOUS DISEASES (2019)

Review Pharmacology & Pharmacy

Recent progress in drug delivery

Chong Li et al.

ACTA PHARMACEUTICA SINICA B (2019)

Article Chemistry, Multidisciplinary

Polymer Dots Compartmentalized in Liposomes as a Photocatalyst for In Situ Hydrogen Therapy

Boyu Zhang et al.

ANGEWANDTE CHEMIE-INTERNATIONAL EDITION (2019)

Article Nanoscience & Nanotechnology

The Onpattro story and the clinical translation of nanomedicines containing nucleic acid-based drugs

Akin Akinc et al.

NATURE NANOTECHNOLOGY (2019)

Review Cell Biology

Functional 5′ UTR mRNA structures in eukaryotic translation regulation and how to find them

Kathrin Leppek et al.

NATURE REVIEWS MOLECULAR CELL BIOLOGY (2018)

Article Pharmacology & Pharmacy

The Use of an Efficient Microfluidic Mixing System for Generating Stabilized Polymeric Nanoparticles for Controlled Drug Release

Yoshinori Morikawa et al.

BIOLOGICAL & PHARMACEUTICAL BULLETIN (2018)

Article Engineering, Biomedical

Noncovalent tagging of siRNA with steroids for transmembrane delivery

Wanyi Tai et al.

BIOMATERIALS (2018)

Article Biophysics

Mechanism of Long-Chain Free Fatty Acid Protonation at the Membrane-Water Interface

Aline A. Pashkovskaya et al.

BIOPHYSICAL JOURNAL (2018)

Article Food Science & Technology

Comparative Effects of Cholesterol and -Sitosterol on the Liposome Membrane Characteristics

Aleksandra A. Jovanovic et al.

EUROPEAN JOURNAL OF LIPID SCIENCE AND TECHNOLOGY (2018)

Article Chemistry, Multidisciplinary

Preclinical evaluation of mRNA trimannosylated lipopolyplexes as therapeutic cancer vaccines targeting dendritic cells

A. Le Moignic et al.

JOURNAL OF CONTROLLED RELEASE (2018)

Article Biotechnology & Applied Microbiology

Self-Amplifying RNA Vaccines Give Equivalent Protection against Influenza to mRNA Vaccines but at Much Lower Doses

Annette B. Vogel et al.

MOLECULAR THERAPY (2018)

Article Nanoscience & Nanotechnology

A modular platform for targeted RNAi therapeutics

Ranit Kedmi et al.

NATURE NANOTECHNOLOGY (2018)

Review Biotechnology & Applied Microbiology

mRNA vaccines - a new era in vaccinology

Norbert Pardi et al.

NATURE REVIEWS DRUG DISCOVERY (2018)

Article Medicine, General & Internal

Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis

D. Adams et al.

NEW ENGLAND JOURNAL OF MEDICINE (2018)

Article Multidisciplinary Sciences

Successful reprogramming of cellular protein production through mRNA delivered by functionalized lipid nanoparticles

Marianna Yanez Arteta et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2018)

Review Immunology

Correlates of adjuvanticity: A review on adjuvants in licensed vaccines

Giuseppe Del Giudice et al.

SEMINARS IN IMMUNOLOGY (2018)

Review Chemistry, Analytical

Progress of Inertial Microfluidics in Principle and Application

Yixing Gou et al.

SENSORS (2018)

Review Biochemistry & Molecular Biology

Lipid Nanoparticles Enabling Gene Therapies: From Concepts to Clinical Utility

Jayesh A. Kulkarni et al.

NUCLEIC ACID THERAPEUTICS (2018)

Article Multidisciplinary Sciences

Cost-effective rapid prototyping and assembly of poly(methyl methacrylate) microfluidic devices

Carlos Matellan et al.

SCIENTIFIC REPORTS (2018)

Article Engineering, Biomedical

mRNA Delivery System for Targeting Antigen-Presenting Cells In Vivo

Cristina Fornaguera et al.

ADVANCED HEALTHCARE MATERIALS (2018)

Review Chemistry, Multidisciplinary

Liposomes and polymersomes: a comparative review towards cell mimicking

Emeline Rideau et al.

CHEMICAL SOCIETY REVIEWS (2018)

Article Chemistry, Multidisciplinary

Non-Viral CRISPR/Cas Gene Editing In Vitro and In Vivo Enabled by Synthetic Nanoparticle Co-Delivery of Cas9 mRNA and sgRNA

Jason B. Miller et al.

ANGEWANDTE CHEMIE-INTERNATIONAL EDITION (2017)

Article Chemistry, Multidisciplinary

Lipid Nanoparticle Assisted mRNA Delivery for Potent Cancer Immunotherapy

Matthias A. Oberli et al.

NANO LETTERS (2017)

Article Chemistry, Multidisciplinary

Lipid Nanoparticles Loaded with an Antisense Oligonucleotide Gapmer Against Bcl-2 for Treatment of Lung Cancer

Xinwei Cheng et al.

PHARMACEUTICAL RESEARCH (2017)

Article Pharmacology & Pharmacy

Novel lipidic and bienzymatic nanosomes for efficient delivery and enhanced bioactivity of catalase

Yao Li et al.

INTERNATIONAL JOURNAL OF PHARMACEUTICS (2017)

Article Biotechnology & Applied Microbiology

Lipid Nanoparticle Systems for Enabling Gene Therapies

Pieter R. Cullis et al.

MOLECULAR THERAPY (2017)

Article Nanoscience & Nanotechnology

Design of lipid nanoparticles for in vitro and in vivo delivery of plasmid DNA

Jayesh A. Kulkarni et al.

NANOMEDICINE-NANOTECHNOLOGY BIOLOGY AND MEDICINE (2017)

Article Multidisciplinary Sciences

Programmable base editing of A.T to G.C in genomic DNA without DNA cleavage

Nicole M. Gaudelli et al.

NATURE (2017)

Review Food Science & Technology

Formulation and application of a new generation of lipid nano-carriers for the food bioactive ingredients

Iman Katouzian et al.

TRENDS IN FOOD SCIENCE & TECHNOLOGY (2017)

Article Biochemistry & Molecular Biology

Molecular Mechanisms of Antisense Oligonucleotides

Stanley T. Crooke

NUCLEIC ACID THERAPEUTICS (2017)

Review Nanoscience & Nanotechnology

Tools for translation: non-viral materials for therapeutic mRNA delivery

Khalid A. Hajj et al.

NATURE REVIEWS MATERIALS (2017)

Article Multidisciplinary Sciences

Dean Flow Dynamics in Low-Aspect Ratio Spiral Microchannels

Nivedita Nivedita et al.

SCIENTIFIC REPORTS (2017)

Article Mathematical & Computational Biology

A systematic analysis of FDA-approved anticancer drugs

Jingchun Sun et al.

BMC SYSTEMS BIOLOGY (2017)

Review Chemistry, Multidisciplinary

Next-Generation Lipids in RNA Interference Therapeutics

Stephanie Rietwyk et al.

ACS NANO (2017)

Article Nanoscience & Nanotechnology

Poly(methyl methacrylate) Surface Modification for Surfactant-Free Real-Time Toxicity Assay on Droplet Microfluidic Platform

Raphael Ortiz et al.

ACS APPLIED MATERIALS & INTERFACES (2017)

Article Engineering, Biomedical

Lipopolyplex potentiates anti-tumor immunity of mRNA-based vaccination

Stefano Persano et al.

BIOMATERIALS (2017)

Article Biochemistry & Molecular Biology

Modified mRNA Vaccines Protect against Zika Virus Infection

Justin M. Richner et al.

Article Pharmacology & Pharmacy

Incorporation of Fenofibrate Nanoparticles Prepared by Melt Emulsification into Polymeric Films

Anagha Bhakay et al.

JOURNAL OF PHARMACEUTICAL INNOVATION (2016)

Article Chemistry, Multidisciplinary

Bioinspired Alkenyl Amino Alcohol Ionizable Lipid Materials for Highly Potent In Vivo mRNA Delivery

Owen S. Fenton et al.

ADVANCED MATERIALS (2016)

Article Biochemical Research Methods

Solid Lipid Nanoparticles for Image-Guided Therapy of Atherosclerosis

Khalid Oumzil et al.

BIOCONJUGATE CHEMISTRY (2016)

Article Biochemical Research Methods

A twisted microfluidic mixer suitable for a wide range of flow rate applications

Shilpa Sivashankar et al.

BIOMICROFLUIDICS (2016)

Article Nanoscience & Nanotechnology

Efficient delivery of Notch1 siRNA to SKOV3 cells by cationic cholesterol derivative-based liposome

Yun-Chun Zhao et al.

INTERNATIONAL JOURNAL OF NANOMEDICINE (2016)

Article Chemistry, Multidisciplinary

Influence of particle size on the in vivo potency of lipid nanoparticle formulations of siRNA

Sam Chen et al.

JOURNAL OF CONTROLLED RELEASE (2016)

Article Medicine, General & Internal

Liposome Delivery Systems for Inhalation: A Critical Review Highlighting Formulation Issues and Anticancer Applications

Mindaugas Rudokas et al.

MEDICAL PRINCIPLES AND PRACTICE (2016)

Review Chemistry, Multidisciplinary

Microfluidic hydrodynamic focusing for synthesis of nanomaterials

Mengqian Lu et al.

NANO TODAY (2016)

Article Multidisciplinary Sciences

Dendrimer-RNA nanoparticles generate protective immunity against lethal Ebola, H1N1 influenza, and Toxoplasma gondii challenges with a single dose

Jasdave S. Chahal et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2016)

Article Medicine, Research & Experimental

Lipid Nanoparticle-mediated siRNA Transfer Against PCTAIRE1/PCTK1/Cdk16 Inhibits In Vivo Cancer Growth

Teruki Yanagi et al.

MOLECULAR THERAPY-NUCLEIC ACIDS (2016)

Editorial Material Pharmacology & Pharmacy

Liposomes in Drug Delivery: How It All Happened

Gregory Gregoriadis

PHARMACEUTICS (2016)

Review Biochemistry & Molecular Biology

Cap-dependent, scanning-free translation initiation mechanisms

Ora Haimov et al.

BIOCHIMICA ET BIOPHYSICA ACTA-GENE REGULATORY MECHANISMS (2015)

Article Chemistry, Medicinal

Effect of Alkyl Chain Length and Unsaturation of the Phospholipid on the Physicochemical Properties of Lipid Nanoparticles

Yuka Funakoshi et al.

CHEMICAL & PHARMACEUTICAL BULLETIN (2015)

Review Chemistry, Multidisciplinary

New Developments in Liposomal Drug Delivery

Bhushan S. Pattni et al.

CHEMICAL REVIEWS (2015)

Article Chemistry, Multidisciplinary

Treatment of neurological disorders by introducing mRNA in vivo using polyplex nanomicelles

Miyuki Baba et al.

JOURNAL OF CONTROLLED RELEASE (2015)

Article Chemistry, Multidisciplinary

Controlled Drug Delivery: Historical perspective for the next generation

Yeon Hee Yun et al.

JOURNAL OF CONTROLLED RELEASE (2015)

Article Chemistry, Physical

Microfluidic Mixing: A General Method for Encapsulating Macromolecules in Lipid Nanoparticle Systems

Alex K. K. Leung et al.

JOURNAL OF PHYSICAL CHEMISTRY B (2015)

Article Materials Science, Biomaterials

Tri-peptide cationic lipids for gene delivery

Yinan Zhao et al.

JOURNAL OF MATERIALS CHEMISTRY B (2015)

Article Biochemistry & Molecular Biology

Cell-penetrating peptide-conjugated lipid nanoparticles for siRNA delivery

Tomohiro Asai et al.

BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS (2014)

Article Agriculture, Multidisciplinary

Enhancing the Stability of Lipid Nanoparticle Systems by Sonication during the Cooling Step and Controlling the Liquid Oil Content

Choongjin Ban et al.

JOURNAL OF AGRICULTURAL AND FOOD CHEMISTRY (2014)

Article Biotechnology & Applied Microbiology

A Cationic Nanoemulsion for the Delivery of Next-generation RNA Vaccines

Luis A. Brito et al.

MOLECULAR THERAPY (2014)

Article Nanoscience & Nanotechnology

In vivo endothelial siRNA delivery using polymeric nanoparticles with low molecular weight

James E. Dahlman et al.

NATURE NANOTECHNOLOGY (2014)

Review Biotechnology & Applied Microbiology

Overcoming the challenges in administering biopharmaceuticals: formulation and delivery strategies

Samir Mitragotri et al.

NATURE REVIEWS DRUG DISCOVERY (2014)

Review Biotechnology & Applied Microbiology

mRNA-based therapeutics - developing a new class of drugs

Ugur Sahin et al.

NATURE REVIEWS DRUG DISCOVERY (2014)

Article Engineering, Chemical

Mixing enhancement of a passive microfluidic mixer containing triangle baffles

Lei Wang et al.

ASIA-PACIFIC JOURNAL OF CHEMICAL ENGINEERING (2014)

Review Chemistry, Multidisciplinary

Materials for Microfluidic Chip Fabrication

Kangning Ren et al.

ACCOUNTS OF CHEMICAL RESEARCH (2013)

Review Pharmacology & Pharmacy

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