☆ 4.8 Review

The landscape of mRNA nanomedicine

NATURE MEDICINE (2022)

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Article Biotechnology & Applied Microbiology

Engineering circular RNA for enhanced protein production

Robert Chen et al.

Summary: In this study, a systematic approach was developed to optimize vector design and enhance protein expression from circRNAs. The optimized circRNAs showed increased translation in vitro, durable translation in vivo, and can be applied to multiple transgenes.

NATURE BIOTECHNOLOGY (2023)

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Article Chemistry, Multidisciplinary

The role of nanoparticle format and route of administration on self-amplifying mRNA vaccine potency

Giulia Anderluzzi et al.

Summary: The efficacy of self-amplifying RNA vaccines is influenced by the delivery route and system format. Intramuscular or intradermal administration of the vaccine initially results in comparable immunogenicity, but ionizable lipid nanoparticles generate higher and long-lasting IgG responses. In contrast, intranasal administration leads to low immune responses and rapid clearance at the administration site.

JOURNAL OF CONTROLLED RELEASE (2022)

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Article Chemistry, Multidisciplinary

Orthogonal Design of Experiments for Optimization of Lipid Nanoparticles for mRNA Engineering of CAR T Cells

Margaret M. Billingsley et al.

Summary: Viral engineered chimeric antigen receptor (CAR) T cell therapies are potent cancer immunotherapies, but their permanent CAR expression can lead to severe adverse effects. Nonviral messenger RNA (mRNA) CAR T cells are being explored as a safer alternative, and this study demonstrates the improved mRNA delivery to T cells with low cytotoxicity using a new ionizable lipid nanoparticle (LNP) formulation (B10). Compared to electroporation, B10 LNPs induce similar CAR expression with reduced cytotoxicity and potent cancer cell killing.

NANO LETTERS (2022)

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Review Chemistry, Multidisciplinary

RNA cancer nanomedicine: nanotechnology-mediated RNA therapy

Bijan Emiliano Ferdows et al.

Summary: This article discusses the research progress in using nanoparticles to deliver therapeutic RNAs for cancer therapy. By selectively regulating cancer-specific genes, researchers have successfully suppressed tumor progression and metastasis, overcoming the degradation of exogenous RNAs by the human immune system. Various types of nanoparticles have been developed for delivering therapeutic RNAs and eliciting tumor suppressing responses.

NANOSCALE (2022)

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Article Multidisciplinary Sciences

Optimization of non-coding regions for a non-modified mRNA COVID-19 vaccine

Makda S. Gebre et al.

Summary: In non-human primates, the second-generation mRNA vaccine CV2CoV was found to induce higher levels of immune responses and provide stronger protection against SARS-CoV-2 variants compared to CVnCoV. The optimization of non-coding regions greatly improved the immunogenicity and protective efficacy of the non-modified mRNA vaccine in non-human primates.

NATURE (2022)

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Article Chemistry, Multidisciplinary

Phase-separating peptides for direct cytosolic delivery and redox-activated release of macromolecular therapeutics

Yue Sun et al.

Summary: This study reports the development of conjugated peptides that can form coacervate microdroplets through liquid-liquid phase separation. These microdroplets have the ability to easily cross the cell membrane and can recruit and release macromolecular therapeutic agents, making them promising for treating various diseases.

NATURE CHEMISTRY (2022)

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Article Biochemical Research Methods

Synthesis of siRNA nanoparticles to silence plaque-destabilizing gene in atherosclerotic lesional macrophages

Xiangang Huang et al.

Summary: Macrophages play a role in promoting plaque progression in atherosclerosis and are an attractive therapeutic target. Researchers have developed siRNA nanoparticles that can silence CaMKII γ activity in macrophages for the treatment of atherosclerosis.

NATURE PROTOCOLS (2022)

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Review Genetics & Heredity

Drug delivery systems for RNA therapeutics

Kalina Paunovska et al.

Summary: This article provides an overview of the importance of RNA-based gene therapy and describes the molecular mechanisms of RNA therapies and different drug delivery systems. It also discusses the path from preclinical research to clinical approval for these therapies.

NATURE REVIEWS GENETICS (2022)

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Article Multidisciplinary Sciences

Intravesical delivery of KDM6A-mRNA via mucoadhesive nanoparticles inhibits the metastasis of bladder cancer

Na Kong et al.

Summary: A mucoadhesive messenger RNA nanoparticle strategy was used for intravesical delivery of KDM6A-mRNA in mice with orthotopic Kdm6a-null BCa, demonstrating KDM6A's therapeutic potential in inhibiting BCa metastasis.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2022)

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Article Multidisciplinary Sciences

CAR T cells produced in vivo to treat cardiac injury

Joel G. Rurik et al.

Summary: This study developed a therapeutic approach to generate antifibrotic CAR T cells in vivo by delivering modified mRNA in T cell-targeted nanoparticles. The efficacy of these in vivo-reprogrammed CAR T cells was evaluated in a mouse model of heart failure.

SCIENCE (2022)

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Article Multidisciplinary Sciences

Combining p53 mRNA nanotherapy with immune checkpoint blockade reprograms the immune microenvironment for effective cancer therapy

Yuling Xiao et al.

Summary: In this study, the authors demonstrate that combining CXCR4-targeted p53 mRNA nanoparticles with anti-PD-1 therapy effectively reverses immunosuppression and improves anti-tumor effects in hepatocellular carcinoma models.

NATURE COMMUNICATIONS (2022)

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Article Multidisciplinary Sciences

In vivo mRNA delivery to virus-specific T cells by light-induced ligand exchange of MHC class I antigen-presenting nanoparticles

Fang-Yi Su et al.

Summary: In this study, we developed Ag-presenting nanoparticles (APNs) for mRNA delivery to multiple populations of antigen-specific CD8(+) T cells using UV-mediated peptide exchange and site-specific conjugation with lipid tails. The data demonstrate the successful transfection of cognate Ag-specific CD8(+) T cells with UV-exchanged APNs.

SCIENCE ADVANCES (2022)

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Article Multidisciplinary Sciences

High-throughput and high-content bioassay enables tuning of polyester nanoparticles for cellular uptake, endosomal escape, and systemic in vivo delivery of mRNA

Yuan Rui et al.

Summary: Nanoparticle-based mRNA therapeutics show great promise, but cellular internalization and endosomal escape pose challenges for cytosolic delivery. Researchers developed a dual nanoparticle uptake and endosomal disruption assay, which can detect cellular internalization and endosomal disruption. The study found that this assay has higher predictive capacity for mRNA delivery compared to conventional polymer and nanoparticle characteristics.

SCIENCE ADVANCES (2022)

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Article Infectious Diseases

Safety and immunogenicity of the SARS-CoV-2 ARCoV mRNA vaccine in Chinese adults: a randomised, double-blind, placebo-controlled, phase 1 trial

Gui-Ling Chen et al.

Summary: This study evaluated the preliminary safety, tolerability, and immunogenicity of an mRNA vaccine (ARCoV) encoding the receptor-binding domain of the SARS-CoV-2 spike protein. The results showed that ARCoV was safe and well-tolerated at all doses and induced strong humoral and cellular immune responses. These findings support further clinical testing of ARCoV on a large scale.

LANCET MICROBE (2022)

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Review Chemistry, Multidisciplinary

Chemo-Enzymatic Modification of the 5? Cap To Study mRNAs

Amarnath Bollu et al.

Summary: The central dogma of molecular biology relies on messenger RNA (mRNA) for the conversion of genetic information. This article highlights the contribution of chemical-enzymatic modification of mRNA at the 5' cap and discusses various methods and applications for modifying mRNA.

ACCOUNTS OF CHEMICAL RESEARCH (2022)

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Article Chemistry, Multidisciplinary

Rapid Surface Display of mRNA Antigens by Bacteria-Derived Outer Membrane Vesicles for a Personalized Tumor Vaccine

Yao Li et al.

Summary: This study utilized bacteria-derived outer membrane vesicles (OMVs) as a delivery platform for mRNA, genetically modified to deliver specific mRNA antigens into dendritic cells, triggering anti-tumor immune responses. The results showed that this platform significantly inhibited tumor progression and achieved 37.5% complete regression in an in vivo model.

ADVANCED MATERIALS (2022)

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Article Immunology

The role of anti-polyethylene glycol (PEG) IgM, IgG, or IgE antibodies in COVID mRNA vaccine anaphylaxis is unknown. We highlight a case with preexisting anti-PEG antibodies that tolerated vaccination

Kristen B. Corey et al.

ANNALS OF ALLERGY ASTHMA & IMMUNOLOGY (2022)

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Article Biochemistry & Molecular Biology

Circular RNA vaccines against SARS-CoV-2 and emerging variants

Liang Qu et al.

Summary: This study reports a circular RNA vaccine that provides potent protection against SARS-CoV-2 by eliciting neutralizing antibodies and T cell responses. The circRNA vaccine exhibits higher and more durable antigen production compared to mRNA vaccines and shows efficacy against the current variants of concern.

CELL (2022)

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Article Chemistry, Multidisciplinary

Rational Design of Bisphosphonate Lipid-like Materials for mRNA Delivery to the Bone Microenvironment

Lulu Xue et al.

Summary: This study developed lipid nanoparticle formulations with a high affinity for the bone microenvironment, which overcame biological barriers and efficiently delivered mRNA therapeutics to the bone microenvironment. The results demonstrated the potential of these formulations for a range of therapeutic applications, including regenerative medicine and gene editing therapies.

JOURNAL OF THE AMERICAN CHEMICAL SOCIETY (2022)

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Review Biotechnology & Applied Microbiology

The clinical progress of mRNA vaccines and immunotherapies

Ann J. Barbier et al.

Summary: The emergency use authorizations (EUAs) of mRNA-based vaccines against SARS-CoV-2 highlight the transformative potential of this nucleic acid technology. Clinical applications of mRNA have mainly focused on vaccines for infectious diseases and cancer, but there are challenges in using mRNA for immunotherapies. The past year has seen significant progress in the clinical development of mRNA therapies for COVID-19, as well as other diseases such as influenza, HIV, and cancer.

NATURE BIOTECHNOLOGY (2022)

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Article Nanoscience & Nanotechnology

Enhancing CRISPR/Cas gene editing through modulating cellular mechanical properties for cancer therapy

Di Zhang et al.

Summary: Modulating the mechanical properties of tumor cells enhances gene editing and inhibits tumor growth by using dendrimer lipid nanoparticles for PD-L1 gene editing and knocking out FAK.

NATURE NANOTECHNOLOGY (2022)

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Article Cell Biology

Adenovirus type 5 SARS-CoV-2 vaccines delivered orally or intranasally reduced disease severity and transmission in a hamster model

Stephanie N. Langel et al.

Summary: Vaccination through oral or intranasal routes can generate strong antibody responses, reduce viral load in the nose and lungs, and mitigate lung pathology caused by SARS-CoV-2. Mucosally vaccinated hamsters transmitted fewer viruses with less severity to naive hamsters, suggesting that mucosal immunization can reduce viral transmission. In a clinical trial, the same platform also induced mucosal cross-reactive antibody responses.

SCIENCE TRANSLATIONAL MEDICINE (2022)

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Article Multidisciplinary Sciences

Combinatorial optimization of mRNA structure, stability, and translation for RNA-based therapeutics

Kathrin Leppek et al.

Summary: The authors developed a platform called PERSIST-seq for studying mRNA stability, ribosome load, and solution stability to improve mRNA therapeutics. They found that in-cell stability is a greater driver of protein output than ribosome load and introduced a method called In-line-seq for mitigating hydrolytic degradation. Their findings demonstrate the simultaneous improvement of mRNA stability and protein expression.

NATURE COMMUNICATIONS (2022)

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Article Multidisciplinary Sciences

Experimental and computational understanding of pulsatile release mechanism from biodegradable core-shell microparticles

Morteza Sarmadi et al.

Summary: This study explores a new microfabrication technique for the fabrication of injectable microparticles with a hollow core-shell structure and pulsatile release kinetics. The sudden increase in porosity of the polymeric matrix leads to the formation of a porous path connecting the core to the environment, enabling pulsatile release. The release kinetics of the microparticles were found to be primarily independent of particle geometry but highly dependent on composition. A qualitative technique was developed to study the pH pattern within the particles, and a computational model successfully simulated the particle deformations before release.

SCIENCE ADVANCES (2022)

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Article Nanoscience & Nanotechnology

Nanotechnology-based strategies against SARS-CoV-2 variants

Xiangang Huang et al.

Summary: This Perspective explores the potential role of nanotechnology in addressing the rise of new SARS-CoV-2 variants. Nanotechnology has been instrumental in the fight against SARS-CoV-2, particularly with the development of highly effective nanotechnology-based mRNA vaccines. However, the emergence of new variants has hindered efforts to bring an end to the pandemic, necessitating the proposal of novel strategies to tackle the crisis posed by existing and emerging SARS-CoV-2 variants.

NATURE NANOTECHNOLOGY (2022)

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Article Materials Science, Multidisciplinary

Inhalable dry powder mRNA vaccines based on extracellular vesicles

Kristen D. Popowski et al.

Summary: Respiratory diseases are a major global burden, and the development of effective therapeutics is limited. Researchers have developed a stable inhalable form of extracellular vesicles that can effectively deliver drugs to the lungs and have shown promising results in vaccine applications.

MATTER (2022)

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Review Chemistry, Multidisciplinary

Emerging mRNA technologies: delivery strategies and biomedical applications

Yufen Xiao et al.

Summary: This article provides an overview of the significant advancements in emerging mRNA technologies, along with the accompanying chemical designs and principles. Various nanoparticle delivery strategies are discussed, and typical mRNA delivery platforms for various biomedical applications are highlighted. The article also offers insights into the challenges and future development of clinical translation for these mRNA technologies.

CHEMICAL SOCIETY REVIEWS (2022)

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Article Biochemistry & Molecular Biology

Intracellular Reverse Transcription of Pfizer BioNTech COVID-19 mRNA Vaccine BNT162b2 In Vitro in Human Liver Cell Line

Markus Alden et al.

Summary: This study investigated the impact of BNT162b2 on human liver cells and found that it rapidly enters the cells and leads to changes in gene expression and distribution.

CURRENT ISSUES IN MOLECULAR BIOLOGY (2022)

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Article Materials Science, Multidisciplinary

Oral mRNA delivery using capsule-mediated gastrointestinal tissue injections

Alex Abramson et al.

Summary: Nucleic acids have opened up new possibilities for therapeutic drugs and vaccines. Oral delivery offers a preferred dosing method and enables local transfection of cells in the gastrointestinal tract. Researchers have developed mRNA nanoparticles and ingestible capsules to achieve transfection in the stomach, demonstrating efficacy in rodents and pigs.

MATTER (2022)

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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)

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Article Chemistry, Multidisciplinary

A Systematic Study of Unsaturation in Lipid Nanoparticles Leads to Improved mRNA Transfection In Vivo

Sang M. Lee et al.

Summary: The study identified an ionizable lipid series (4A3) with a citronellol-based (Cit) periphery as the best performer out of 91 amino lipids, showing superior lipid fusion and improved mRNA delivery efficacy in vivo through Selective ORgan Targeting (SORT). These findings demonstrate how lipid unsaturation promotes mRNA delivery and how lipid mixing can enhance efficacy.

ANGEWANDTE CHEMIE-INTERNATIONAL EDITION (2021)

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News Item Biotechnology & Applied Microbiology

Exosome-based candidates move into the clinic

Megan Cully

Summary: Exosomes, small extracellular vesicles shed by cells, have long been considered promising drug delivery systems for various biologic payloads, with the first ones now in clinical trials.

NATURE REVIEWS DRUG DISCOVERY (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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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)

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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)

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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 Immunology

Nanomaterial Delivery Systems for mRNA Vaccines

Michael D. Buschmann et al.

Summary: This review highlights the recent success of mRNA vaccines in SARS-CoV-2 clinical trials, emphasizing the crucial role of lipid nanoparticles in the delivery systems and providing an analysis of the determinants of lipid nanoparticles' performance in mRNA vaccines.

VACCINES (2021)

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Article Chemistry, Multidisciplinary

Targeted Delivery of mRNA with One-Component Ionizable Amphiphilic Janus Dendrimers

Dapeng Zhang et al.

Summary: Efficient delivery of nucleic acids is crucial in genetic nanomedicine, and lipid nanoparticles as well as Janus dendrimers have shown great success in delivering mRNA for Covid-19 vaccines. By modifying the structure, targeted delivery of mRNA using Janus dendrimers can be switched from lung to liver and spleen, expanding the utility of dendrimersome nanoparticles from therapeutics to vaccines.

JOURNAL OF THE AMERICAN CHEMICAL SOCIETY (2021)

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Article Chemistry, Multidisciplinary

Selective Encapsulation of Therapeutic mRNA in Engineered Extracellular Vesicles by DNA Aptamer

Siyan Zhang et al.

Summary: The study proposed an efficient strategy to load therapeutic mRNAs into EVs for effective gene therapy. The mRNA delivered through this strategy showed significant therapeutic effects both in vitro and in vivo, indicating its broad potential application in the field of gene therapy.

NANO LETTERS (2021)

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Article Chemistry, Multidisciplinary

Intercalation-Driven Formation of siRNA Nanogels for Cancer Therapy

Xiangang Huang et al.

Summary: This study presents a novel approach using an intercalation-driven siRNA nanogels (NG) as a delivery vehicle, which demonstrates good stability and efficient intracellular delivery for cancer therapy.

NANO LETTERS (2021)

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Article Multidisciplinary Sciences

An ultra-low-cost electroporator with microneedle electrodes (ePatch) for SARS-CoV-2 vaccination

Dengning Xia et al.

Summary: The novel ePatch system enables low-cost, handheld DNA vaccination against SARS-CoV-2, inducing strong antibody responses with at least 10-fold dose sparing compared to conventional injection methods. The vaccination process is well tolerated with mild, transient effects on the skin, offering a portable and accessible approach for combating COVID-19 and other epidemics.

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

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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)

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Article Chemistry, Multidisciplinary

Insights from nanotechnology in COVID-19 treatment

Zhongmin Tang et al.

Summary: In response to the COVID-19 pandemic, nanotechnology plays a significant role in antiviral research, particularly in drug and vaccine delivery, interference in the virus life cycle, and pneumonia treatment.

NANO TODAY (2021)

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Article Biotechnology & Applied Microbiology

Treatment of influenza and SARS-CoV-2 infections via mRNA-encoded Cas13a in rodents

Emmeline L. Blanchard et al.

Summary: Cas13a delivered to the lung of mice and hamsters inhibits replication of influenza virus and SARS-CoV-2, suggesting that Cas13a-mediated targeting of pathogenic viruses can mitigate respiratory infections. The study successfully reduced viral RNA levels in the lung tissue of animals, demonstrating the efficacy of Cas13a in targeting RNA viruses.

NATURE BIOTECHNOLOGY (2021)

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Article Chemistry, Physical

Membrane-destabilizing ionizable phospholipids for organ-selective mRNA delivery and CRISPR-Cas gene editing

Shuai Liu et al.

Summary: The study successfully delivered mRNA or single-guide RNA for gene editing in vivo using combinatorially synthesized ionizable phospholipids. Optimized iPhos lipids can change shapes in endosomal acidic environments to facilitate the release of gene editing materials. When working synergistically with helper lipids, iPhos lipids can achieve targeted delivery to specific organs.

NATURE MATERIALS (2021)

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Article Multidisciplinary Sciences

Delivery of mRNA vaccine with a lipid-like material potentiates antitumor efficacy through Toll-like receptor 4 signaling

Hongxia Zhang et al.

Summary: The C1 lipid nanoparticle-based mRNA cancer nanovaccine efficiently delivers mRNA to antigen presenting cells, activates TLR4, induces inflammatory cytokines in dendritic cells, and shows significant antitumor efficacy.

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

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Article Cell Biology

mRNA vaccination induces tick resistance and prevents transmission of the Lyme disease agent

Andaleeb Sajid et al.

Summary: The study showed that vaccination with 19ISP mRNA can enhance host recognition of tick bites, reduce tick engorgement, and prevent B. burgdorferi infection. Immunization with 19ISP also hindered the transmission of the pathogen in vaccinated guinea pigs.

SCIENCE TRANSLATIONAL MEDICINE (2021)

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Article Multidisciplinary Sciences

Biomimetic nanoparticles deliver mRNAs encoding costimulatory receptors and enhance T cell mediated cancer immunotherapy

Wenqing Li et al.

Summary: The study demonstrates that delivering costimulatory receptor mRNA to tumor-infiltrating T cells enhances the antitumor effects of antibodies, leading to significantly improved antitumor activity in multiple tumor models. This combination strategy could boost the antitumor immune response and has the potential to enhance cancer immunotherapy.

NATURE COMMUNICATIONS (2021)

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Article Multidisciplinary Sciences

Engineered ionizable lipid nanoparticles for targeted delivery of RNA therapeutics into different types of cells in the liver

M. Kim et al.

Summary: Engineered LNPs have been developed for targeted delivery of RNA to hepatocytes and LSECs, showing potential for cell type-specific delivery of RNA to the liver and other tissues.

SCIENCE ADVANCES (2021)

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Article Chemistry, Multidisciplinary

Chemo-Enzymatic Modification of the 5′ Cap Maintains Translation and Increases Immunogenic Properties of mRNA

Melissa van Duelmen et al.

Summary: Eukaryotic mRNAs can be modified with site-specific chemo-enzymatic methods to enhance immune response, offering a potential strategy for future therapeutic applications.

ANGEWANDTE CHEMIE-INTERNATIONAL EDITION (2021)

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Article Biotechnology & Applied Microbiology

A single dose of self-transcribing and replicating RNA-based SARS-CoV-2 vaccine produces protective adaptive immunity in mice

Ruklanthi de Alwis et al.

Summary: The LUNAR-COV19 vaccine has been successfully developed to induce strong antibody response and cell-mediated immune response in mice. The vaccine at different doses can completely protect mice from SARS-CoV-2 challenge.

MOLECULAR THERAPY (2021)

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Article Multidisciplinary Sciences

In vivo CRISPR base editing of PCSK9 durably lowers cholesterol in primates

Kiran Musunuru et al.

Summary: This study demonstrates the efficient and precise modification of disease-related genes in living cynomolgus monkeys using CRISPR base editors delivered in vivo via lipid nanoparticles. The results provide proof-of-concept for the potential therapeutic application of CRISPR base editors in making precise single-nucleotide changes in target genes, supporting a 'once-and-done' approach for the treatment of diseases.

NATURE (2021)

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Article Biotechnology & Applied Microbiology

In vivo adenine base editing of PCSK9 in macaques reduces LDL cholesterol levels

Tanja Rothgangl et al.

Summary: ABEs have been shown to effectively and safely reduce LDL levels in the livers of mice and cynomolgus macaques. Using lipid nanoparticle-based delivery of mRNA encoding an ABE and a sgRNA targeting PCSK9, significant reductions in PCSK9 and LDL levels were achieved in both species. Further research is warranted to explore the potential of ABEs in treating monogenic liver diseases.

NATURE BIOTECHNOLOGY (2021)

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Article Multidisciplinary Sciences

Reverse-transcribed SARS-CoV-2 RNA can integrate into the genome of cultured human cells and can be expressed in patient-derived tissues

Liguo Zhang et al.

Summary: Researchers have found that SARS-CoV-2 RNA may be reverse-transcribed and integrated into the DNA of human cells, leading to positive PCR results in patients even after infection and recovery. However, the integrated viral sequences mainly consist of subgenomic sequences, which cannot produce infectious virus.

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

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Review Immunology

mRNA vaccines manufacturing: Challenges and bottlenecks

Sara Sousa Rosa et al.

Summary: mRNA vaccines are emerging as a new alternative to conventional vaccines, with their precision, safety profile, and flexible manufacturing. They have been used to combat the Covid-19 pandemic and are being studied for other diseases as well. However, challenges in manufacturing, technology platform, and cost-effective production need to be addressed to fully utilize this new vaccination technology.

VACCINE (2021)

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Article Immunology

Dissolvable Microneedle Patches to Enable Increased Access to Vaccines against SARS-CoV-2 and Future Pandemic Outbreaks

Jesse O'Shea et al.

Summary: The dissolvable microneedle patches offer multiple advantages for vaccination, including improved immunogenicity, dose-sparing effects, cost reduction, waste reduction, and simplified supply chain. They have great potential to improve vaccination globally and save lives in the context of COVID-19 and future pandemic outbreaks.

VACCINES (2021)

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Review Pharmacology & Pharmacy

Nucleic acid delivery for therapeutic applications

Akash Gupta et al.

Summary: This review discusses various therapeutic applications of nucleic acids targeted at specific diseases and tissues, highlighting recent technologies that have advanced to late-stage clinical trials and received FDA approval.

ADVANCED DRUG DELIVERY REVIEWS (2021)

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Article Engineering, Biomedical

Systemic delivery of mRNA and DNA to the lung using polymer-lipid nanoparticles*

James C. Kaczmarek et al.

Summary: Non-viral vectors can deliver nucleic acids into cells in vivo, with study showing that PBAEs with altered monomer chemistry can effectively deliver DNA or mRNA to lung endothelium in mice. Physical properties like particle size or charge do not explain differences in transfection efficacy, indicating tailored delivery vectors are needed for different nucleic acids. In vivo luciferase expression was significantly higher with mRNA compared to pDNA, highlighting the potential of PBAEs as intracellular delivery materials.

BIOMATERIALS (2021)

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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)

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Article Chemistry, Multidisciplinary

One-Component Multifunctional Sequence-Defined Ionizable Amphiphilic Janus Dendrimer Delivery Systems for mRNA

Dapeng Zhang et al.

Summary: Efficient delivery of nucleic acids is crucial for genetic nanomedicine. While viral vectors are highly efficient, they can also cause severe immune responses, toxicity, and mutations. Nonviral lipid nanoparticles containing specific components show promise for mRNA delivery, but challenges such as stability at low temperatures need to be addressed.

JOURNAL OF THE AMERICAN CHEMICAL SOCIETY (2021)

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Article Biotechnology & Applied Microbiology

Highly efficient CD4+T cell targeting and genetic recombination using engineered CD4+cell-homing mRNA-LNPs

Istvan Tombacz et al.

Summary: The study demonstrates that conjugating CD4 antibody to LNPs enables specific targeting and mRNA interventions to CD4+ cells (including T cells), providing a platform technology for immunotherapy of devastating conditions and HIV cure.

MOLECULAR THERAPY (2021)

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Review Biotechnology & Applied Microbiology

mRNA vaccines for infectious diseases: principles, delivery and clinical translation

Namit Chaudhary et al.

Summary: mRNA vaccines have been established as a rapid, effective, and safe approach for protecting individuals from infectious diseases, especially during the COVID-19 pandemic. Further research is needed to optimize mRNA vaccine design, delivery, and applications for a range of infectious diseases.

NATURE REVIEWS DRUG DISCOVERY (2021)

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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)

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Article Infectious Diseases

Efficacy and safety of the CVnCoV SARS-CoV-2 mRNA vaccine candidate in ten countries in Europe and Latin America (HERALD): a randomised, observer-blinded, placebo-controlled, phase 2b/3 trial

Peter G Kremsner et al.

LANCET INFECTIOUS DISEASES (2021)

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Letter Multidisciplinary Sciences

No evidence of SARS-CoV-2 reversetranscription and integration as the origin of chimeric transcripts in patient tissues

Rhys Parry et al.

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

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Article Multidisciplinary Sciences

Mammalian retrovirus-like protein PEG10 packages its own mRNA and can be pseudotyped for mRNA delivery

Michael Segel et al.

Summary: Eukaryotic genomes contain domesticated genes from integrating viruses and mobile genetic elements, including mammalian Gag homologs and PEG10, which can be used for RNA delivery. The development of selective endogenous encapsidation for cellular delivery (SEND) using PEG10 demonstrates its potential as an efficient therapeutic delivery modality.

SCIENCE (2021)

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Article Cell Biology

Reactivation of the tumor suppressor PTEN by mRNA nanoparticles enhances antitumor immunity in preclinical models

Yao-Xin Lin et al.

Summary: Clinical evidence suggests that restoration of functional PTEN through mRNA delivery by polymeric nanoparticles can effectively induce immune activation in tumor cells, reversing immunosuppressive tumor microenvironment and enhancing sensitivity to immune checkpoint blockade therapy. In vivo results show that PTEN mRNA nanoparticles promote CD8(+) T cell infiltration, increase proinflammatory cytokine expression, and decrease regulatory T cells and myeloid-derived suppressor cells, resulting in potent antitumor effects and immunological memory in cancer models.

SCIENCE TRANSLATIONAL MEDICINE (2021)

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Article Multidisciplinary Sciences

High-throughput 5′ UTR engineering for enhanced protein production in non-viral gene therapies

Jicong Cao et al.

Summary: By using a high-throughput screening approach on 5'UTR libraries, this study identified three synthetic 5'UTRs that outperformed commonly used non-viral gene therapy plasmids in expressing protein payloads. This finding has important implications for engineered cell and gene therapies.

NATURE COMMUNICATIONS (2021)

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Article Multidisciplinary Sciences

Nucleoside-modified VEGFC mRNA induces organ-specific lymphatic growth and reverses experimental lymphedema

Daniel Szoke et al.

Summary: Dysfunction of the lymphatic system leads to secondary lymphedema and decreases quality of life. Researchers demonstrate that delivery of nucleoside-modified Vascular Endothelial Growth Factor C (VEGFC) mRNA induces organ-specific lymphatic growth and reverses experimental lymphedema.

NATURE COMMUNICATIONS (2021)

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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)

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Article Chemistry, Multidisciplinary

An mRNA SARS-CoV-2 Vaccine Employing Charge-Altering Releasable Transporters with a TLR-9 Agonist Induces Neutralizing Antibodies and T Cell Memory

Ole A. W. Haabeth et al.

Summary: The success of two FDA-approved mRNA vaccines in combating the SARS-CoV-2 pandemic has demonstrated the speed and therapeutic potential of mRNA vaccines. To ensure flexibility in future vaccine development, different vaccination strategies and alternative mRNA delivery vehicles are desirable.

ACS CENTRAL SCIENCE (2021)

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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)

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Article Immunology

Immunologic mechanisms of seasonal influenza vaccination administered by microneedle patch from a randomized phase I trial

Nadine G. Rouphael et al.

Summary: The study found that inactivated influenza virus vaccination through dissolvable microneedle patches (MNPs) produces humoral and cellular immune responses that are similar or greater than traditional intramuscular (IM) vaccination. MNPs induced higher neuraminidase inhibition (NAI) titers for all three influenza virus strains tested and stimulated a larger percentage of circulating T follicular helper cells.

NPJ VACCINES (2021)

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Article Biochemistry & Molecular Biology

Intranasal delivery of replicating mRNA encoding neutralizing antibody against SARS-CoV-2 infection in mice

Jia-Qi Li et al.

Summary: The study demonstrated a safe and cost-effective method to prevent SARS-CoV-2 infection by expressing neutralizing antibodies in the lungs. Through in vitro and in vivo assays, it was shown that self-replicating mRNA could efficiently block SARS-CoV-2 infection with reduced viral titer and tissue damage in the lung with the help of lung target delivery using the VRP system.

SIGNAL TRANSDUCTION AND TARGETED THERAPY (2021)

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Review Oncology

Recent advances and discoveries in the mechanisms and functions of CAR T cells

Rebecca C. Larson et al.

Summary: This review summarizes the major advances that have been made in the efficacy and safety of CAR T cell therapies over the past 3 years and looks ahead to new findings that will impact the future of this immunotherapy.

NATURE REVIEWS CANCER (2021)

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Article Engineering, Biomedical

Adenine base editing in an adult mouse model of tyrosinaemia

Chun-Qing Song et al.

NATURE BIOMEDICAL ENGINEERING (2020)

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Article Chemistry, Multidisciplinary

Mild Innate Immune Activation Overrides Efficient Nanoparticle-Mediated RNA Delivery

Melissa P. Lokugamage et al.

ADVANCED MATERIALS (2020)

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Article Pharmacology & Pharmacy

Pharmacokinetics of Patisiran, the First Approved RNA Interference Therapy in Patients With Hereditary Transthyretin-Mediated Amyloidosis

Xiaoping Zhang et al.

JOURNAL OF CLINICAL PHARMACOLOGY (2020)

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Review Biochemistry & Molecular Biology

Tailoring mRNA Vaccine to Balance Innate/Adaptive Immune Response

Sergio Linares-Fernandez et al.

TRENDS IN MOLECULAR MEDICINE (2020)

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Article Chemistry, Multidisciplinary

A Combinatorial Library of Lipid Nanoparticles for RNA Delivery to Leukocytes

Srinivas Ramishetti et al.

ADVANCED MATERIALS (2020)

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Article Chemistry, Multidisciplinary

Ionizable Lipid Nanoparticle-Mediated mRNA Delivery for Human CAR T Cell Engineering

Margaret M. Billingsley et al.

NANO LETTERS (2020)

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Article Engineering, Biomedical

Large-scale generation of functional mRNA-encapsulating exosomes via cellular nanoporation

Zhaogang Yang et al.

NATURE BIOMEDICAL ENGINEERING (2020)

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Article Multidisciplinary Sciences

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

Lei Miao et al.

NATURE COMMUNICATIONS (2020)

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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)

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Review Pharmacology & Pharmacy

Anti-PEG antibodies: Properties, formation, testing and role in adverse immune reactions to PEGylated nano-biopharmaceuticals

Gergely Tibor Kozma et al.

ADVANCED DRUG DELIVERY REVIEWS (2020)

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Article Biochemistry & Molecular Biology

A Thermostable mRNA Vaccine against COVID-19

Na-Na Zhang et al.

CELL (2020)

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Article Multidisciplinary Sciences

Impact of mRNA chemistry and manufacturing process on innate immune activation

Jennifer Nelson et al.

SCIENCE ADVANCES (2020)

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Article Multidisciplinary Sciences

Increased PIP3 activity blocks nanoparticle mRNA delivery

Kalina Paunovska et al.

SCIENCE ADVANCES (2020)

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Article Chemistry, Multidisciplinary

Leveraging mRNA Sequences and Nanoparticles to Deliver SARS-CoV-2 Antigens In Vivo

Chunxi Zeng et al.

ADVANCED MATERIALS (2020)

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Editorial Material Multidisciplinary Sciences

Hacking the body's delivery service

Amanda B. Keener

NATURE (2020)

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Article Multidisciplinary Sciences

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

Ugur Sahin et al.

NATURE (2020)

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Article Cell Biology

Engineered PLGA microparticles for long-term, pulsatile release of STING agonist for cancer immunotherapy

Xueguang Lu et al.

SCIENCE TRANSLATIONAL MEDICINE (2020)

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Article Cell Biology

siRNA nanoparticles targeting CaMKIIγ in lesional macrophages improve atherosclerotic plaque stability in mice

Wei Tao et al.

SCIENCE TRANSLATIONAL MEDICINE (2020)

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Article Multidisciplinary Sciences

Self-amplifying RNA SARS-CoV-2 lipid nanoparticle vaccine candidate induces high neutralizing antibody titers in mice

Paul F. McKay et al.

NATURE COMMUNICATIONS (2020)

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Review Nanoscience & Nanotechnology

A materials-science perspective on tackling COVID-19

Zhongmin Tang et al.

NATURE REVIEWS MATERIALS (2020)

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Article Multidisciplinary Sciences

In vitro-transcribed antigen receptor mRNA nanocarriers for transient expression in circulating T cells in vivo

N. N. Parayath et al.

NATURE COMMUNICATIONS (2020)

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Article Multidisciplinary Sciences

Engineered mutant α-ENaC subunit mRNA delivered by lipid nanoparticles reduces amiloride currents in cystic fibrosis-based cell and mice models

Anindit Mukherjee et al.

SCIENCE ADVANCES (2020)

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Article Materials Science, Multidisciplinary

Efficient Delivery of mRNA Using Crosslinked Nucleic Acid Nanogel as a Carrier

Xiangang Huang et al.

ACS MATERIALS LETTERS (2020)

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Article Multidisciplinary Sciences

CRISPR-Cas9 genome editing using targeted lipid nanoparticles for cancer therapy

Daniel Rosenblum et al.

SCIENCE ADVANCES (2020)

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Article Biochemistry & Molecular Biology

RNA Circularization Diminishes Immunogenicity and Can Extend Translation Duration In Vivo

R. Alexander Wesselhoeft et al.

MOLECULAR CELL (2019)

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Review Biotechnology & Applied Microbiology

mRNA as a Transformative Technology for Vaccine Development to Control Infectious Diseases

Giulietta Maruggi et al.

MOLECULAR THERAPY (2019)

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Review Biotechnology & Applied Microbiology

Delivering the Messenger: Advances in Technologies for Therapeutic mRNA Delivery

Piotr S. Kowalski et al.

MOLECULAR THERAPY (2019)

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Article Cell Biology

Durable anticancer immunity from intratumoral administration of IL-23, IL-36γ, and OX40L mRNAs

Susannah L. Hewitt et al.

SCIENCE TRANSLATIONAL MEDICINE (2019)

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Article Chemistry, Multidisciplinary

Oligo(serine ester) Charge-Altering Releasable Transporters: Organocatalytic Ring-Opening Polymerization and their Use for in Vitro and in Vivo mRNA Delivery

Nancy L. Benner et al.

JOURNAL OF THE AMERICAN CHEMICAL SOCIETY (2019)

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Article Medicine, Research & Experimental

Optimization of Lipid Nanoparticles for Intramuscular Administration of mRNA Vaccines

Kimberly J. Hassett et al.

MOLECULAR THERAPY-NUCLEIC ACIDS (2019)

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Article Medicine, Research & Experimental

A Facile Method for the Removal of dsRNA Contaminant from In Vitro-Transcribed mRNA

Markus Baiersdoerfer et al.

MOLECULAR THERAPY-NUCLEIC ACIDS (2019)

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Article Biotechnology & Applied Microbiology

Human 5′ UTR design and variant effect prediction from a massively parallel translation assay

Paul J. Sample et al.

NATURE BIOTECHNOLOGY (2019)

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Article Biotechnology & Applied Microbiology

Delivery of mRNA vaccines with heterocyclic lipids increases anti-tumor efficacy by STING-mediated immune cell activation

Lei Miao et al.

NATURE BIOTECHNOLOGY (2019)

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Article Multidisciplinary Sciences

Linkage between endosomal escape of LNP-mRNA and loading into EVs for transport to other cells

Marco Maugeri et al.

NATURE COMMUNICATIONS (2019)

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Article Chemistry, Multidisciplinary

Inhaled Nanoformulated mRNA Polyplexes for Protein Production in Lung Epithelium

Asha Kumari Patel et al.

ADVANCED MATERIALS (2019)

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Review Biochemistry & Molecular Biology

mRNA as novel technology for passive immunotherapy

Thomas Schlake et al.

CELLULAR AND MOLECULAR LIFE SCIENCES (2019)

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Article Biotechnology & Applied Microbiology

Improving mRNA-Based Therapeutic Gene Delivery by Expression-Augmenting 3′ UTRs Identified by Cellular Library Screening

Alexandra G. Orlandini von Niessen et al.

MOLECULAR THERAPY (2019)

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Article Cell Biology

Synthetic mRNA nanoparticle-mediated restoration of p53 tumor suppressor sensitizes p53-deficient cancers to mTOR inhibition

Na Kong et al.

SCIENCE TRANSLATIONAL MEDICINE (2019)

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Article Chemistry, Multidisciplinary

Nanoparticles Containing Oxidized Cholesterol Deliver mRNA to the Liver Microenvironment at Clinically Relevant Doses

Kalina Paunovska et al.

ADVANCED MATERIALS (2019)

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Review Biophysics

Controlling the movement of molecules

Robert Langer

QUARTERLY REVIEWS OF BIOPHYSICS (2019)

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Article Pathology

Safety Evaluation of Lipid Nanoparticle-Formulated Modified mRNA in the Sprague-Dawley Rat and Cynomolgus Monkey

Maja Sedic et al.

VETERINARY PATHOLOGY (2018)

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Article Immunology

Nucleoside-modified mRNA vaccines induce potent T follicular helper and germinal center B cell responses

Norbert Pardi et al.

JOURNAL OF EXPERIMENTAL MEDICINE (2018)

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Article Biotechnology & Applied Microbiology

A Novel Amino Lipid Series for mRNA Delivery: Improved Endosomal Escape and Sustained Pharmacology and Safety in Non-human Primates

Staci Sabnis et al.

MOLECULAR THERAPY (2018)

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Article Nanoscience & Nanotechnology

A modular platform for targeted RNAi therapeutics

Ranit Kedmi et al.

NATURE NANOTECHNOLOGY (2018)

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Review Biotechnology & Applied Microbiology

mRNA vaccines - a new era in vaccinology

Norbert Pardi et al.

NATURE REVIEWS DRUG DISCOVERY (2018)

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Article Multidisciplinary Sciences

Enhanced mRNA delivery into lymphocytes enabled by lipid-varied libraries of charge-altering releasable transporters

Colin J. McKinlay et al.

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

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Article Multidisciplinary Sciences

Engineering circular RNA for potent and stable translation in eukaryotic cells

R. Alexander Wesselhoeft et al.

NATURE COMMUNICATIONS (2018)

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Article Cell Biology

A Single Administration of CRISPR/Cas9 Lipid Nanoparticles Achieves Robust and Persistent In Vivo Genome Editing

Jonathan D. Finn et al.

CELL REPORTS (2018)

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Article Toxicology

Correlation of the cytotoxic effects of cationic lipids with their headgroups

Shaohui Cui et al.

TOXICOLOGY RESEARCH (2018)

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Article Chemistry, Multidisciplinary

Optimization of a Degradable Polymer-Lipid Nanoparticle for Potent Systemic Delivery of mRNA to the Lung Endothelium and Immune Cells

James C. Kaczmarek et al.

NANO LETTERS (2018)

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Article Multidisciplinary Sciences

mRNA vaccination with charge-altering releasable transporters elicits human T cell responses and cures established tumors in mice

Ole A. W. Haabeth et al.

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

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Article Engineering, Biomedical

Restoration of tumour-growth suppression in vivo via systemic nanoparticle-mediated delivery of PTEN mRNA

Mohammad Ariful Islam et al.

NATURE BIOMEDICAL ENGINEERING (2018)

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Article Biochemistry & Molecular Biology

Systemic messenger RNA as an etiological treatment for acute intermittent porphyria

Lei Jiang et al.

NATURE MEDICINE (2018)

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Article Multidisciplinary Sciences

Cell specific delivery of modified mRNA expressing therapeutic proteins to leukocytes

Nuphar Veiga et al.

NATURE COMMUNICATIONS (2018)

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Article Biochemistry & Molecular Biology

Translation of CircRNAs

Nagarjuna Reddy Pamudurti et al.

MOLECULAR CELL (2017)

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Article Biochemistry & Molecular Biology

Circ-ZNF609 Is a Circular RNA that Can Be Translated and Functions in Myogenesis

Ivano Legnini et al.

MOLECULAR CELL (2017)

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Article Biotechnology & Applied Microbiology

Lipid Nanoparticle Systems for Enabling Gene Therapies

Pieter R. Cullis et al.

MOLECULAR THERAPY (2017)

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Article Multidisciplinary Sciences

Personalized RNA mutanome vaccines mobilize poly-specific therapeutic immunity against cancer

Ugur Sahin et al.

NATURE (2017)

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Article Multidisciplinary Sciences

Zika virus protection by a single low-dose nucleoside-modified mRNA vaccination

Norbert Pardi et al.

NATURE (2017)

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Article Biotechnology & Applied Microbiology

Structure-guided chemical modification of guide RNA enables potent non-viral in vivo genome editing

Hao Yin et al.

NATURE BIOTECHNOLOGY (2017)

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Editorial Material Genetics & Heredity

Translated circular RNAs

Linda Koch

NATURE REVIEWS GENETICS (2017)

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Editorial Material Medicine, General & Internal

A Milestone for CAR T Cells

Eric Tran et al.

NEW ENGLAND JOURNAL OF MEDICINE (2017)

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Article Multidisciplinary Sciences

Charge-altering releasable transporters (CARTs) for the delivery and release of mRNA in living animals

Colin J. McKinlay et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2017)

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Article Multidisciplinary Sciences

Fabrication of fillable microparticles and other complex 3D microstructures

Kevin J. McHugh et al.

SCIENCE (2017)

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Review Immunology

Delivering the right message: Challenges and opportunities in lipid nanoparticles-mediated modified mRNA therapeutics-An innate immune system standpoint

Yasmin Granot et al.

SEMINARS IN IMMUNOLOGY (2017)

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Article Multidisciplinary Sciences

Hit-and-run programming of therapeutic cytoreagents using mRNA nanocarriers

H. F. Moffett et al.

NATURE COMMUNICATIONS (2017)

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Article Multidisciplinary Sciences

Administration of nucleoside-modified mRNA encoding broadly neutralizing antibody protects humanized mice from HIV-1 challenge

Norbert Pardi et al.

NATURE COMMUNICATIONS (2017)

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Review Nanoscience & Nanotechnology

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

Khalid A. Hajj et al.

NATURE REVIEWS MATERIALS (2017)

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Article Immunology

Unmodified mRNA in LNPs constitutes a competitive technology for prophylactic vaccines

Johannes Lutz et al.

NPJ VACCINES (2017)

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Article Biochemistry & Molecular Biology

Vaccine Mediated Protection Against Zika Virus-Induced Congenital Disease

Justin M. Richner et al.

CELL (2017)

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Article Cardiac & Cardiovascular Systems

Safety With Echocardiographic Contrast Agents

Preetham R. Muskula et al.

CIRCULATION-CARDIOVASCULAR IMAGING (2017)

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Article Biochemistry & Molecular Biology

Modified mRNA Vaccines Protect against Zika Virus Infection

Justin M. Richner et al.

CELL (2017)

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Article Biochemistry & Molecular Biology

Circular RNAs are long-lived and display only minimal early alterations in response to a growth factor

Yehoshua Enuka et al.

NUCLEIC ACIDS RESEARCH (2016)

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Review Pharmacology & Pharmacy

The role of helper lipids in lipid nanoparticles (LNPs) designed for oligonucleotide delivery

Xinwei Cheng et al.

ADVANCED DRUG DELIVERY REVIEWS (2016)

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Review Engineering, Biomedical

Effect of surface properties on liposomal siRNA delivery

Yuqiong Xia et al.

BIOMATERIALS (2016)

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Review Pharmacology & Pharmacy

Evading innate immunity in nonviral mRNA delivery: don't shoot the messenger

Joke Devoldere et al.

DRUG DISCOVERY TODAY (2016)

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Article Chemistry, Multidisciplinary

Materials for non-viral intracellular delivery of messenger RNA therapeutics

Kevin J. Kauffman et al.

JOURNAL OF CONTROLLED RELEASE (2016)

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Article Multidisciplinary Sciences

Systemic RNA delivery to dendritic cells exploits antiviral defence for cancer immunotherapy

Lena M. Kranz et al.

NATURE (2016)

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Article Biochemistry & Molecular Biology

mRNA capping: biological functions and applications

Anand Ramanathan et al.

NUCLEIC ACIDS RESEARCH (2016)

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Editorial Material Biochemistry & Molecular Biology

Who discovered messenger RNA?

Matthew Cobb

CURRENT BIOLOGY (2015)

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Article Chemistry, Multidisciplinary

N1-methylpseudouridine-incorporated mRNA outperforms pseudouridine-incorporated mRNA by providing enhanced protein expression and reduced immunogenicity in mammalian cell lines and mice

Oliwia Andries et al.

JOURNAL OF CONTROLLED RELEASE (2015)

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Article Biotechnology & Applied Microbiology

Sequence-engineered mRNA Without Chemical Nucleoside Modifications Enables an Effective Protein Therapy in Large Animals

Andreas Thess et al.

MOLECULAR THERAPY (2015)

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Article Biochemistry & Molecular Biology

Toll-like receptor 8 senses degradation products of single-stranded RNA

Hiromi Tanji et al.

NATURE STRUCTURAL & MOLECULAR BIOLOGY (2015)

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Article Biochemistry & Molecular Biology

Exon-intron circular RNAs regulate transcription in the nucleus

Zhaoyong Li et al.

NATURE STRUCTURAL & MOLECULAR BIOLOGY (2015)

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Article Multidisciplinary Sciences

Long-circulating siRNA nanoparticles for validating Prohibitin1-targeted non-small cell lung cancer treatment

Xi Zhu et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2015)

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Article Biochemistry & Molecular Biology

Toll-like receptor 8 senses degradation products of single-stranded RNA

Hiromi Tanji et al.

NATURE STRUCTURAL & MOLECULAR BIOLOGY (2015)

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Article Biochemistry & Molecular Biology

Exon-intron circular RNAs regulate transcription in the nucleus

Zhaoyong Li et al.

NATURE STRUCTURAL & MOLECULAR BIOLOGY (2015)

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Article Biochemistry & Molecular Biology

An RNA Editor, Adenosine Deaminase Acting on Double-Stranded RNA (ADAR1)

Cyril X. George et al.

JOURNAL OF INTERFERON AND CYTOKINE RESEARCH (2014)

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Review Biotechnology & Applied Microbiology

mRNA-based therapeutics - developing a new class of drugs

Ugur Sahin et al.

NATURE REVIEWS DRUG DISCOVERY (2014)

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Review Cell Biology

Trafficking of endosomal Toll-like receptors

Bettina L. Lee et al.

TRENDS IN CELL BIOLOGY (2014)

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Article Biotechnology & Applied Microbiology

Biodegradable Lipids Enabling Rapidly Eliminated Lipid Nanoparticles for Systemic Delivery of RNAi Therapeutics

Martin A. Maier et al.

MOLECULAR THERAPY (2013)

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Article Multidisciplinary Sciences

Circular RNAs are a large class of animal RNAs with regulatory potency

Sebastian Memczak et al.

NATURE (2013)

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Article Biotechnology & Applied Microbiology

Increased Erythropoiesis in Mice Injected With Submicrogram Quantities of Pseudouridine-containing mRNA Encoding Erythropoietin

Katalin Kariko et al.

MOLECULAR THERAPY (2012)

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Article Multidisciplinary Sciences

Nonviral delivery of self-amplifying RNA vaccines

Andrew J. Geall et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2012)

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Article Biotechnology & Applied Microbiology

Expression of therapeutic proteins after delivery of chemically modified mRNA in mice

Michael S. D. Kormann et al.

NATURE BIOTECHNOLOGY (2011)

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Article Immunology

Ribose 2′-O-methylation provides a molecular signature for the distinction of self and non-self mRNA dependent on the RNA sensor Mda5

Roland Zuest et al.

NATURE IMMUNOLOGY (2011)

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Article Biochemistry & Molecular Biology

Generating the optimal mRNA for therapy: HPLC purification eliminates immune activation and improves translation of nucleoside-modified, protein-encoding mRNA

Katalin Kariko et al.

NUCLEIC ACIDS RESEARCH (2011)

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Article Biochemistry & Molecular Biology

Nucleoside modifications in RNA limit activation of 2'-5'-oligoadenylate synthetase and increase resistance to cleavage by RNase L

Bart R. Anderson et al.

NUCLEIC ACIDS RESEARCH (2011)

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Article Chemistry, Multidisciplinary

mRNA transfection of cervical carcinoma and mesenchymal stem cells mediated by cationic carriers

Joanna Rejman et al.

JOURNAL OF CONTROLLED RELEASE (2010)

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Article Biochemistry & Molecular Biology

Incorporation of pseudouridine into mRNA enhances translation by diminishing PKR activation

Bart R. Anderson et al.

NUCLEIC ACIDS RESEARCH (2010)

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Article Biochemistry & Molecular Biology

The Centrality of RNA

Phillip A. Sharp

CELL (2009)

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Article Immunology

Recognition of 5′ Triphosphate by RIG-I Helicase Requires Short Blunt Double-Stranded RNA as Contained in Panhandle of Negative-Strand Virus

Martin Schlee et al.

IMMUNITY (2009)

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Review Chemistry, Multidisciplinary

A combinatorial polymer library approach yields insight into nonviral gene delivery

Jordan J. Green et al.

ACCOUNTS OF CHEMICAL RESEARCH (2008)

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Article Biotechnology & Applied Microbiology

Incorporation of Pseudouridine Into mRNA Yields Superior Nonimmunogenic Vector With Increased Translational Capacity and Biological Stability

Katalin Kariko et al.

MOLECULAR THERAPY (2008)

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Article Multidisciplinary Sciences

Structural basis of toll-like receptor 3 signaling with double-stranded RNA

Lin Liu et al.

SCIENCE (2008)

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Review Virology

Viral encounters with 2′,5′-oligoadenylate synthetase and RNase L during the interferon antiviral response

Robert H. Silverman

JOURNAL OF VIROLOGY (2007)

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Review Biochemistry & Molecular Biology

The dsRNA protein kinase PKR:: Virus and cell control

M. A. Garcia et al.

BIOCHIMIE (2007)

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Article Hematology

Modification of antigen-encoding RNA increases stability, translational efficacy, and T-cell stimulatory capacity of dendritic cells

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Review Chemistry, Multidisciplinary

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Review Immunology

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Article Immunology

Suppression of RNA recognition by Toll-like receptors:: The impact of nucleoside modification and the evolutionary origin of RNA

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Article Multidisciplinary Sciences

Innate antiviral responses by means of TLR7-mediated recognition of single-stranded RNA

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Article Multidisciplinary Sciences

Species-specific recognition of single-stranded RNA via toll-like receptor 7 and 8

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Article Chemistry, Multidisciplinary

Parallel synthesis and biophysical characterization of a degradable polymer library for gene delivery

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JOURNAL OF THE AMERICAN CHEMICAL SOCIETY (2003)

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Article Biochemistry & Molecular Biology

A new class of fusion-associated small transmembrane (FAST) proteins encoded by the non-enveloped fusogenic reoviruses

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