4.7 Review

Recent Advances in Lipid Nanoparticles for Delivery of mRNA

Related references

Note: Only part of the references are listed.
Review Pharmacology & Pharmacy

Lipid nanomaterials-based RNA therapy and cancer treatment

Xingcai Zhang et al.

Summary: This article summarizes the most important advances in RNA delivery and nanomedicine, introducing lipid nanoparticle-based RNA therapeutics and their impact on drug development. It describes the fundamental properties of key RNA members and recent advances in nanoparticles for targeted RNA delivery, with a focus on lipid nanoparticles. The review also presents recent advances in biomedical therapy using RNA drug delivery and state-of-the-art RNA application platforms, including the treatment of different types of cancer.

ACTA PHARMACEUTICA SINICA B (2023)

Article Chemistry, Multidisciplinary

Liposome-based anchoring and core-encapsulation for combinatorial cancer therapy

Qingqing Xiao et al.

Summary: In this study, a liposome-based co-delivery system was developed to simultaneously deliver pro-apoptotic protein and cytotoxic drug for synergistic cancer treatment. The system improved the solubility of the drug and achieved efficient drug delivery by targeting receptors on cancer cells. In vivo experiments demonstrated that the co-delivery system significantly inhibited tumor growth.

CHINESE CHEMICAL LETTERS (2022)

Review Biochemical Research Methods

Principles for designing an optimal mRNA lipid nanoparticle vaccine

Edo Kon et al.

Summary: mRNA lipid nanoparticles (LNPs) have gained significant attention as therapeutic platforms due to the success of the SARS-CoV-2 mRNA LNP vaccines. The modularity and rapid manufacturing capabilities are the advantages of mRNA-LNP vaccines. When designing optimal mRNA-LNPs, choices regarding efficacy, stability, and toxicity need to be made.

CURRENT OPINION IN BIOTECHNOLOGY (2022)

Article Chemistry, Multidisciplinary

Microfluidic technologies and devices for lipid nanoparticle-based RNA delivery

Masatoshi Maeki et al.

Summary: mRNA vaccines have been approved for COVID-19 prevention, thanks to the development of RNA-delivery technology such as lipid nanoparticles (LNPs). Recently, microfluidic devices and technologies have been utilized for the production of RNA-loaded LNPs, providing advantages such as precise control over LNP size, reproducibility, and high-throughput optimization.

JOURNAL OF CONTROLLED RELEASE (2022)

Article Multidisciplinary Sciences

Lung-selective mRNA delivery of synthetic lipid nanoparticles for the treatment of pulmonary lymphangioleiomyomatosis

Min Qiu et al.

Summary: The challenge of delivering mRNA to specific organs and cells in vivo is being addressed, with research showing that N-series LNPs can selectively deliver mRNA to the lungs while O-series LNPs tend to deliver mRNA to the liver. By analyzing the protein corona on targeted LNPs, unique plasma proteins were identified that contribute to their targetability. This research demonstrates the potential of LNPs as a promising therapeutic intervention for lung diseases like lymphangioleiomyomatosis.

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

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)

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)

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)

Article Chemistry, Multidisciplinary

Liposome-based delivery of biological drugs

Kosheli Thapa Magar et al.

Summary: Biological drugs are limited in their application due to their inherent properties, but liposome-based drug delivery systems provide a promising solution to improve drug delivery.

CHINESE CHEMICAL LETTERS (2022)

Article Biochemistry & Molecular Biology

Humoral and cellular immune memory to four COVID-19 vaccines

Zeli Zhang et al.

Summary: Multiple COVID-19 vaccines have successfully protected against symptomatic cases and deaths. Comparisons of T cell, B cell, and antibody responses to different vaccines can provide insights into protective immunity against COVID-19, particularly immune memory. mRNA vaccines and Ad26.COV2.S induced strong T cell responses, while mRNA vaccines showed substantial declines in antibodies.
Article Polymer Science

Effective mRNA Protection by Poly(l-ornithine) Synergizes with Endosomal Escape Functionality of a Charge-Conversion Polymer toward Maximizing mRNA Introduction Efficiency

Anjaneyulu Dirisala et al.

Summary: This study integrates the protective effect against nucleases and the endosomal escape capability into a single polyplex by fine-tuning the catiomer chemical structure and incorporating the endosomal escape modality for efficient mRNA delivery. The methylene spacer length on the catiomer side chain significantly affects the nuclease stability of the polyplex, and introducing a charge-conversion polymer can improve endosomal escape efficiency by approximately 80-fold in cultured cells. The system synergizes PLO's protective effect against nucleases and CCP's endosomal escape capability, leading to enhanced protein expression efficiency from mRNA delivery.

MACROMOLECULAR RAPID COMMUNICATIONS (2022)

Article Immunology

IL-1 and IL-1ra are key regulators of the inflammatory response to RNA vaccines

Siri Tahtinen et al.

Summary: Lipid-formulated RNA vaccines can cause systemic inflammatory responses in humans, which were not predicted in preclinical studies. The IL-1 pathway plays a key role in triggering vaccine-induced inflammation, but this effect is dampened in mice.

NATURE IMMUNOLOGY (2022)

Article Medicine, General & Internal

Effectiveness of heterologous and homologous covid-19 vaccine regimens: living systematic review with network meta-analysis

Wing Ying Au et al.

Summary: The study evaluates the effectiveness of heterologous and homologous COVID-19 vaccine regimens in preventing COVID-19 infections, hospital admissions, and death. Results show that a three-dose mRNA vaccine regimen is most effective against asymptomatic and symptomatic COVID-19 infections, while a homologous two-dose mRNA regimen is highly effective in preventing severe COVID-19 infections.

BMJ-BRITISH MEDICAL JOURNAL (2022)

Review Pharmacology & Pharmacy

mRNA cancer vaccines: Advances, trends and challenges

Qing He et al.

Summary: mRNA vaccines are well-tolerated by patients and offer flexibility in the choice of encoded molecules. The approval of the first mRNA-based COVID-19 vaccine by the FDA in 2021 has sparked enthusiasm for mRNA vaccine research and development. mRNA cancer vaccines have become a hot topic and have undergone rapid development in recent years.

ACTA PHARMACEUTICA SINICA B (2022)

Article Chemistry, Multidisciplinary

Stronger together for in-cell translation: natural and unnatural base modified mRNA

Lisa Bornewasser et al.

Summary: In this study, we report the in-cell application of highly modified mRNA using in vitro transcription and visualization of their distribution in cells through click chemistry. By incorporating natural base modifications and site-specifically introduced artificial nucleotides, we were able to improve the translation efficiency of mRNA while maintaining its functionality. This approach provides a new way to enhance mRNA therapeutics.

CHEMICAL SCIENCE (2022)

Review Chemistry, Multidisciplinary

Lipid Nanoparticle-mRNA Formulations for Therapeutic Applications

Chang Wang et al.

Summary: This article highlights the effectiveness of lipid nanoparticles in delivering mRNA and their applications in treating various diseases. It also focuses on the recent advances in designing and developing different classes of lipids and lipid derivatives, as well as research directions in mRNA engineering.

ACCOUNTS OF CHEMICAL RESEARCH (2021)

Review Pharmacology & Pharmacy

Difference in the lipid nanoparticle technology employed in three approved siRNA (Patisiran) and mRNA (COVID-19 vaccine) drugs

Yuta Suzuki et al.

Summary: Nucleic acid therapeutics are evolving into precise medicines that can manipulate specific genes, with the challenge of developing safe and effective delivery systems for target cells. Lipid nanoparticles (LNPs) have provided a revolutionary delivery system allowing for the clinical translation of RNA-based candidates. LNPs enabled the development of two SARS-CoV-2 mRNA vaccines during the COVID-19 outbreak in 2020.

DRUG METABOLISM AND PHARMACOKINETICS (2021)

Review Chemistry, Analytical

Analytical characterization of liposomes and other lipid nanoparticles for drug delivery

Yuchen Fan et al.

Summary: Lipid nanoparticles, including liposomes and lipid/nucleic acid complexed nanoparticles, have shown success in the pharmaceutical industry due to stable drug loading, extended pharmacokinetics, reduced off-target side effects, and enhanced delivery efficiency to disease targets. Analytical characterization is crucial for drug design, formulation development, and quality control.

JOURNAL OF PHARMACEUTICAL AND BIOMEDICAL ANALYSIS (2021)

Article Biotechnology & Applied Microbiology

Targeting herpes simplex virus with CRISPR-Cas9 cures herpetic stromal keratitis in mice

Di Yin et al.

Summary: The study targeted HSV-1 genomes directly using mRNA-carrying lentiviral particles (HELP), which efficiently blocked HSV-1 replication and reduced the occurrence of HSK in three different infection models. HELP was capable of eliminating the viral reservoir via retrograde transport from corneas to trigeminal ganglia, while inhibiting viral replication in human-derived corneas without causing off-target effects.

NATURE BIOTECHNOLOGY (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 Multidisciplinary Sciences

Murine liver repair via transient activation of regenerative pathways in hepatocytes using lipid nanoparticle-complexed nucleoside-modified mRNA

Fatima Rizvi et al.

Summary: The study introduces a potentially translatable therapeutic intervention for liver regeneration, utilizing mRNA-LNP delivery to induce hepatocyte proliferation and accelerate liver function recovery in murine models of acute and chronic liver injury.

NATURE COMMUNICATIONS (2021)

Article Allergy

mRNA Vaccines to Prevent COVID-19 Disease and Reported Allergic Reactions: Current Evidence and Suggested Approach

Aleena Banerji et al.

Summary: The FDA has authorized emergency use of Pfizer-BioNTech and Moderna COVID-19 vaccines, but individuals with severe allergic reactions are advised against receiving them. The CDC recommends observing those with a history of immediate allergies or anaphylaxis for 30 minutes post-vaccination, while all others should be observed for 15 minutes.

JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY-IN PRACTICE (2021)

Article Engineering, Biomedical

Lentiviral delivery of co-packaged Cas9 mRNA and a Vegfa-targeting guide RNA prevents wet age-related macular degeneration in mice

Sikai Ling et al.

Summary: The study demonstrates the efficacy of engineered lentiviruses co-packaging SpCas9 mRNA and a guide RNA targeting the Vegfa gene in preventing wet age-related macular degeneration in mice, providing a potential new treatment approach for retinal neovascular diseases.

NATURE BIOMEDICAL ENGINEERING (2021)

Article Chemistry, Multidisciplinary

mRNA loading into ATP-responsive polyplex micelles with optimal density of phenylboronate ester crosslinking to balance robustness in the biological milieu and intracellular translational efficiency

Naoto Yoshinaga et al.

Summary: A novel carrier design was introduced to protect and release mRNA in the cytosol for efficient protein expression, balancing stability in the biological milieu and ATP-responsive release through the modulation of structural factors. Furthermore, the optimal formulation was enhanced by incorporating cholesterol moieties for prolonged blood circulation post intravenous injection.

JOURNAL OF CONTROLLED RELEASE (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 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)

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)

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

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)

Review Chemistry, Multidisciplinary

The progress and perspective of strategies to improve tumor penetration of nanomedicines

Jiang Hu et al.

Summary: This article discusses the importance of tumor penetration, analyzes the barriers to tumor penetration, and discusses strategies such as modulating the tumor microenvironment to enhance tumor penetration.

CHINESE CHEMICAL LETTERS (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 Chemistry, Multidisciplinary

Impact of lipid nanoparticle size on mRNA vaccine immunogenicity

Kimberly J. Hassett et al.

Summary: The size of LNP particles was found to affect the immunogenicity of vaccines in mice, with smaller diameter LNPs being less immunogenic; however, all particle sizes tested yielded robust immune responses in non-human primates (NHP).

JOURNAL OF CONTROLLED RELEASE (2021)

Article Biochemistry & Molecular Biology

Solid Lipid Nanoparticles Produced via a Coacervation Method as Promising Carriers for Controlled Release of Quercetin

Luigi Talarico et al.

Summary: Quercetin-loaded Solid Lipid Nanoparticles were successfully synthesized in this study, showing high encapsulation efficiency and suitable controlled release. The samples were characterized qualitatively using various techniques, and the encapsulation efficiency, drug release, and antioxidant effect were evaluated in vitro.

MOLECULES (2021)

Article Nanoscience & Nanotechnology

Conformation-sensitive targeting of lipid nanoparticles for RNA therapeutics

Niels Dammes et al.

Summary: The study demonstrates successful in vivo gene silencing in a selective subset of leukocytes using a conformation-sensitive targeting strategy, showing potential therapeutic applications in a murine model of colitis. The results suggest that this lipid nanoparticle targeting strategy could be applied for selective delivery of payloads to other conformation-sensitive targets.

NATURE NANOTECHNOLOGY (2021)

Article Biochemistry & Molecular Biology

Theoretical basis for stabilizing messenger RNA through secondary structure design

Hannah K. Wayment-Steele et al.

Summary: RNA hydrolysis poses challenges for mRNA-based vaccines and therapeutics due to issues in manufacturing, long-term storage, global delivery, and in vivo stability. Redesigning RNAs to form double-stranded regions could reduce hydrolysis, with algorithms like those on the Eterna platform showing promise in creating 'superfolder' mRNAs with increased stability and diverse features beneficial for translation and immunogenicity. Ongoing research aims to maximize mRNA stability and longevity through innovative design strategies and computational approaches.

NUCLEIC ACIDS RESEARCH (2021)

Article Mathematical & Computational Biology

Are the Allergic Reactions of COVID-19 Vaccines Caused by mRNA Constructs or Nanocarriers? Immunological Insights

Gurudeeban Selvaraj et al.

Summary: FDA has authorized BNT162b2 and mRNA-1273 vaccines for emergency use against COVID-19, but severe allergic reactions have been reported during vaccination, leading to exploration of potential causes of anaphylaxis following vaccination.

INTERDISCIPLINARY SCIENCES-COMPUTATIONAL LIFE SCIENCES (2021)

Review Pharmacology & Pharmacy

Protamine-Based Strategies for RNA Transfection

Natalia Teresa Jarzebska et al.

Summary: Protamine is a natural cationic peptide mixture used for heparin neutralization and slow-release insulin formulations. It is also employed for cellular delivery of nucleic acids. Protamine plays a dual role in RNA stabilization, protecting against degradation and enhancing cell penetration.

PHARMACEUTICS (2021)

Article Toxicology

Why are we vaccinating children against COVID-19?

Ronald N. Kostoff et al.

Summary: The article highlights concerns regarding COVID-19 inoculations for children, noting that most deaths attributed to COVID-19 occur in the elderly while children have low COVID-19 mortality rates. Post-inoculation deaths are mainly seen in elderly individuals, with children also experiencing some deaths. The short-term clinical trials for the vaccines did not adequately represent the total population, especially adolescents and children, and failed to consider long-term effects or changes in biomarkers. Additionally, a cost-benefit analysis suggests that the number of deaths attributed to each inoculation may outweigh the deaths from COVID-19 in the most vulnerable 65+ demographic.

TOXICOLOGY REPORTS (2021)

Article Engineering, Biomedical

Optimization of lipid nanoparticles for the delivery of nebulized therapeutic mRNA to the lungs

Melissa P. Lokugamage et al.

Summary: This study optimized the design of lipid nanoparticles for the efficient delivery of therapeutic mRNA to the lungs via nebulization, resulting in protection of mice from a lethal challenge of H1N1 influenza A virus and more efficient delivery of mRNA compared to LNPs previously optimized for systemic delivery. The cluster approach to LNP design may facilitate the optimization of LNPs for other administration routes and therapeutics.

NATURE BIOMEDICAL ENGINEERING (2021)

Review Chemistry, Multidisciplinary

Exosome-mediated delivery of gene vectors for gene therapy

Li Duan et al.

Summary: Gene vectors deliver genetic materials or gene editing devices into cells for therapeutic protein production or gene correction, but barriers like cell membrane and degradation necessitate carriers for clinical gene therapy. Exosomes, engineered to encapsulate and deliver nucleic acids, and their fusion with liposomes increase loading capacity and maintain targeting capability, showing promise for future gene delivery applications in therapy.

NANOSCALE (2021)

Review Pharmacology & Pharmacy

Dendrimers in the context of nanomedicine

Ana Paula Dias et al.

INTERNATIONAL JOURNAL OF PHARMACEUTICS (2020)

Review Chemistry, Multidisciplinary

The role of liposomes in clinical nanomedicine development. What now? Now what?

Daan J. A. Crommelin et al.

JOURNAL OF CONTROLLED RELEASE (2020)

Article Chemistry, Multidisciplinary

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

Margaret M. Billingsley et al.

NANO LETTERS (2020)

Article Multidisciplinary Sciences

Selective targeting of nanomedicine to inflamed cerebral vasculature to enhance the blood-brain barrier

Oscar A. Marcos-Contreras et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2020)

Review Multidisciplinary Sciences

The biology, function, and biomedical applications of exosomes

Raghu Kalluri et al.

SCIENCE (2020)

Review Pharmacology & Pharmacy

Opportunities and Challenges in the Delivery of mRNA-Based Vaccines

Abishek Wadhwa et al.

PHARMACEUTICS (2020)

Article Engineering, Biomedical

Lipid-Modified Aminoglycosides for mRNA Delivery to the Liver

Xueliang Yu et al.

ADVANCED HEALTHCARE MATERIALS (2020)

Article Cell & Tissue Engineering

Naturally Derived Membrane Lipids Impact Nanoparticle-Based Messenger RNA Delivery

Jeonghwan Kim et al.

CELLULAR AND MOLECULAR BIOENGINEERING (2020)

Article Medicine, Research & Experimental

Intranasal Delivery and Transfection of mRNA Therapeutics in the Brain Using Cationic Liposomes

Harkiranpreet Kaur Dhaliwal et al.

MOLECULAR PHARMACEUTICS (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)

Review Pharmacology & Pharmacy

Lipid nanoparticles for nucleic acid delivery: Current perspectives

Eleni Samaridou et al.

ADVANCED DRUG DELIVERY REVIEWS (2020)

Review Pharmacology & Pharmacy

Lipid nanoparticle technology for therapeutic gene regulation in the liver

Dominik Witzigmann et al.

ADVANCED DRUG DELIVERY REVIEWS (2020)

Article Chemistry, Multidisciplinary

Imidazole-Based Synthetic Lipidoids for In Vivo mRNA Delivery into Primary T Lymphocytes

Xuewei Zhao et al.

ANGEWANDTE CHEMIE-INTERNATIONAL EDITION (2020)

Review Immunology

Recent advances in mRNA vaccine technology

Norbert Pardi et al.

CURRENT OPINION IN IMMUNOLOGY (2020)

Article Engineering, Biomedical

Long-term storage of lipid-like nanoparticles for mRNA delivery

Pengxuan Zhao et al.

BIOACTIVE MATERIALS (2020)

Article Biotechnology & Applied Microbiology

Nanoparticles containing constrained phospholipids deliver mRNA to liver immune cells in vivo without targeting ligands

Zubao Gan et al.

BIOENGINEERING & TRANSLATIONAL MEDICINE (2020)

Article Nanoscience & Nanotechnology

Polysarcosine-Functionalized Lipid Nanoparticles for Therapeutic mRNA Delivery

Sara S. Nogueira et al.

ACS APPLIED NANO MATERIALS (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)

Article Materials Science, Biomaterials

Adjuvant incorporated lipid nanoparticles for enhanced mRNA-mediated cancer immunotherapy

Kyuri Lee et al.

BIOMATERIALS SCIENCE (2020)

Review Biotechnology & Applied Microbiology

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

Giulietta Maruggi et al.

MOLECULAR THERAPY (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 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)

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 Biochemistry & Molecular Biology

Transfection Efficiency Evaluation and Endocytosis Exploration of Different Polymer Condensed Agents

Jianhua Wang et al.

DNA AND CELL BIOLOGY (2019)

Article Multidisciplinary Sciences

mRNA structure regulates protein expression through changes in functional half-life

David M. Mauger et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2019)

Article Chemistry, Multidisciplinary

Ionizable lipid nanoparticles encapsulating barcoded mRNA for accelerated in vivo delivery screening

Pedro P. G. Guimaraes et al.

JOURNAL OF CONTROLLED RELEASE (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)

Article Chemistry, Multidisciplinary

On the role of helper lipids in lipid nanoparticle formulations of siRNA

Jayesh A. Kulkarni et al.

NANOSCALE (2019)

Review Pharmacology & Pharmacy

Brief update on endocytosis of nanomedicines

Siddharth Patel et al.

ADVANCED DRUG DELIVERY REVIEWS (2019)

Review Cell Biology

Codon optimality, bias and usage in translation and mRNA decay

Gavin Hanson et al.

NATURE REVIEWS MOLECULAR CELL BIOLOGY (2018)

Review Biotechnology & Applied Microbiology

Gene therapy clinical trials worldwide to 2017: An update

Samantha L. Ginn et al.

JOURNAL OF GENE MEDICINE (2018)

Article Chemistry, Physical

Structure of Lipid Nanoparticles Containing siRNA or mRNA by Dynamic Nuclear Polarization-Enhanced NMR Spectroscopy

Jasmine Viger-Gravel et al.

JOURNAL OF PHYSICAL CHEMISTRY B (2018)

Article Medicine, Research & Experimental

Serum Nuclease Susceptibility of mRNA Cargo in Condensed Polyplexes

Albert Yen et al.

MOLECULAR PHARMACEUTICS (2018)

Article Nanoscience & Nanotechnology

A modular platform for targeted RNAi therapeutics

Ranit Kedmi et al.

NATURE NANOTECHNOLOGY (2018)

Review Multidisciplinary Sciences

Gene therapy comes of age

Cynthia E. Dunbar et al.

SCIENCE (2018)

Article Biochemistry & Molecular Biology

Metabolically stabilized double-stranded mRNA polyplexes

Jacob A. Poliskey et al.

GENE THERAPY (2018)

Article Biochemistry & Molecular Biology

Scaffold-mediated delivery for non-viral mRNA vaccines

Ruying Chen et al.

GENE THERAPY (2018)

Review Nanoscience & Nanotechnology

Main trends of immune effects triggered by nanomedicines in preclinical studies

Blanka Halamoda-Kenzaoui et al.

INTERNATIONAL JOURNAL OF NANOMEDICINE (2018)

Review Chemistry, Multidisciplinary

Biomaterials for vaccine-based cancer immunotherapy

Rui Zhang et al.

JOURNAL OF CONTROLLED RELEASE (2018)

Article Multidisciplinary Sciences

High-throughput in vivo screen of functional mRNA delivery identifies nanoparticles for endothelial cell gene editing

Cory D. Sago et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (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 Nanoscience & Nanotechnology

Achieving long-term stability of lipid nanoparticles: examining the effect of pH, temperature, and lyophilization

Rebecca L. Ball et al.

INTERNATIONAL JOURNAL OF NANOMEDICINE (2017)

Article Chemistry, Multidisciplinary

Lipid Nanoparticle Assisted mRNA Delivery for Potent Cancer Immunotherapy

Matthias A. Oberli et al.

NANO LETTERS (2017)

Review Biotechnology & Applied Microbiology

The Future Looks Brighter After 25 Years of Retinal Gene Therapy

Alberto Auricchio et al.

HUMAN GENE THERAPY (2017)

Article Chemistry, Multidisciplinary

Boosting Intracellular Delivery of Lipid Nanoparticle-Encapsulated mRNA

Siddharth Patel et al.

NANO LETTERS (2017)

Article Multidisciplinary Sciences

Structure of human IFIT1 with capped RNA reveals adaptable mRNA binding and mechanisms for sensing N1 and N2 ribose 2′-O methylations

Yazan M. Abbas et al.

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

Article Engineering, Biomedical

Lipopolyplex potentiates anti-tumor immunity of mRNA-based vaccination

Stefano Persano et al.

BIOMATERIALS (2017)

Article Pharmacology & Pharmacy

Applications of lipid nanoparticles in gene therapy

Ana del Pozo-Rodriguez et al.

EUROPEAN JOURNAL OF PHARMACEUTICS AND BIOPHARMACEUTICS (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)

Editorial Material Biochemistry & Molecular Biology

Who discovered messenger RNA?

Matthew Cobb

CURRENT BIOLOGY (2015)

Review Nanoscience & Nanotechnology

Polymeric Nanocarriers for Non-Viral Gene Delivery

Ling Li et al.

JOURNAL OF BIOMEDICAL NANOTECHNOLOGY (2015)

Article Chemistry, Multidisciplinary

Expression kinetics of nucleoside-modified mRNA delivered in lipid nanoparticles to mice by various routes

Norbert Pardi et al.

JOURNAL OF CONTROLLED RELEASE (2015)

Editorial Material Genetics & Heredity

A deno-associated virus finds its disease

David W. Russell et al.

NATURE GENETICS (2015)

Article Biotechnology & Applied Microbiology

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

Luis A. Brito et al.

MOLECULAR THERAPY (2014)

Review Genetics & Heredity

Non-viral vectors for gene-based therapy

Hao Yin et al.

NATURE REVIEWS GENETICS (2014)

Article Multidisciplinary Sciences

Broad and adaptable RNA structure recognition by the human interferon-induced tetratricopeptide repeat protein IFIT5

George E. Katibah et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2014)

Article Medicine, Research & Experimental

RNA Interference by Single-and Double-stranded siRNA With a DNA Extension Containing a 3′ Nuclease-resistant Mini-hairpin Structure

Simon J. Allison et al.

MOLECULAR THERAPY-NUCLEIC ACIDS (2014)

Article Medicine, Research & Experimental

Shielding of Lipid Nanoparticles for siRNA Delivery: Impact on Physicochemical Properties, Cytokine Induction, and Efficacy

Varun Kumar et al.

MOLECULAR THERAPY-NUCLEIC ACIDS (2014)

Review Pharmacology & Pharmacy

Liposomal drug delivery systems: From concept to clinical applications

Theresa M. Allen et al.

ADVANCED DRUG DELIVERY REVIEWS (2013)

Article Genetics & Heredity

History of gene therapy

Thomas Wirth et al.

Article Chemistry, Multidisciplinary

Transfection efficiency and transgene expression kinetics of mRNA delivered in naked and nanoparticle format

Kyle K. L. Phua et al.

JOURNAL OF CONTROLLED RELEASE (2013)

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)

Article Biotechnology & Applied Microbiology

Image-based analysis of lipid nanoparticle-mediated siRNA delivery, intracellular trafficking and endosomal escape

Jerome Gilleron et al.

NATURE BIOTECHNOLOGY (2013)

Article Immunology

Rapidly produced SAM® vaccine against H7N9 influenza is immunogenic in mice

Armin Hekele et al.

EMERGING MICROBES & INFECTIONS (2013)

Article Pharmacology & Pharmacy

Advances in Lipid Nanoparticles for siRNA Delivery

Yuen Tam et al.

Pharmaceutics (2013)

Review Chemistry, Multidisciplinary

Nucleic Acid Delivery: The Missing Pieces of the Puzzle?

Juliane Nguyen et al.

ACCOUNTS OF CHEMICAL RESEARCH (2012)

Article Chemistry, Multidisciplinary

Maximizing the Potency of siRNA Lipid Nanoparticles for Hepatic Gene Silencing In Vivo

Muthusamy Jayaraman et al.

ANGEWANDTE CHEMIE-INTERNATIONAL EDITION (2012)

Review Pharmacology & Pharmacy

Factors Controlling Nanoparticle Pharmacokinetics: An Integrated Analysis and Perspective

S. M. Moghimi et al.

ANNUAL REVIEW OF PHARMACOLOGY AND TOXICOLOGY, VOL 52 (2012)

Review Pharmacology & Pharmacy

Lipid nanoparticles: state of the art, new preparation methods and challenges in drug delivery

Luigi Battaglia et al.

EXPERT OPINION ON DRUG DELIVERY (2012)

Article Biotechnology & Applied Microbiology

Protective efficacy of in vitro synthesized, specific mRNA vaccines against influenza A virus infection

Benjamin Petsch et al.

NATURE BIOTECHNOLOGY (2012)

Article Multidisciplinary Sciences

A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity

Martin Jinek et al.

SCIENCE (2012)

Article Medicine, Research & Experimental

Microfluidic Synthesis of Highly Potent Limit-size Lipid Nanoparticles for In Vivo Delivery of siRNA

Nathan M. Belliveau et al.

MOLECULAR THERAPY-NUCLEIC ACIDS (2012)

Article Polymer Science

Effect of Chain Length on Cytotoxicity and Endocytosis of Cationic Polymers

Jinge Cai et al.

MACROMOLECULES (2011)

Article Biotechnology & Applied Microbiology

Targeted Delivery of RNAi Therapeutics With Endogenous and Exogenous Ligand-Based Mechanisms

Akin Akinc et al.

MOLECULAR THERAPY (2010)

Article Biotechnology & Applied Microbiology

Rational design of cationic lipids for siRNA delivery

Sean C. Semple et al.

NATURE BIOTECHNOLOGY (2010)

Article Biochemistry & Molecular Biology

Incorporation of pseudouridine into mRNA enhances translation by diminishing PKR activation

Bart R. Anderson et al.

NUCLEIC ACIDS RESEARCH (2010)

Review Pharmacology & Pharmacy

Degradable polyethylenimines as DNA and small interfering RNA carriers

D. Jere et al.

EXPERT OPINION ON DRUG DELIVERY (2009)

Article Biotechnology & Applied Microbiology

A combinatorial library of lipid-like materials for delivery of RNAi therapeutics

Akin Akinc et al.

NATURE BIOTECHNOLOGY (2008)

Article Biotechnology & Applied Microbiology

Renal clearance of quantum dots

Hak Soo Choi et al.

NATURE BIOTECHNOLOGY (2007)

Article Anatomy & Morphology

Contribution of high-resolution correlative imaging techniques in the study of the liver sieve in three-dimensions

Filip Braet et al.

MICROSCOPY RESEARCH AND TECHNIQUE (2007)

Article Multidisciplinary Sciences

RNAi-mediated gene silencing in non-human primates

TS Zimmermann et al.

NATURE (2006)

Article Biochemistry & Molecular Biology

Stabilized plasmid-lipid particles containing PEG-diacylglycerols exhibit extended circulation lifetimes and tumor selective gene expression

E Ambegia et al.

BIOCHIMICA ET BIOPHYSICA ACTA-BIOMEMBRANES (2005)

Article Pharmacology & Pharmacy

Immunogenicity and rapid blood clearance of liposomes containing polyethylene glycol-lipid conjugates and nucleic acid

SC Semple et al.

JOURNAL OF PHARMACOLOGY AND EXPERIMENTAL THERAPEUTICS (2005)

Article Chemistry, Multidisciplinary

A scalable, extrusion-free method for efficient liposomal encapsulation of plasmid DNA

LB Jeffs et al.

PHARMACEUTICAL RESEARCH (2005)

Article Multidisciplinary Sciences

LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1

S Hacein-Bey-Abina et al.

SCIENCE (2003)

Article Biochemistry & Molecular Biology

On the mechanism whereby cationic lipids promote intracellular delivery of polynucleic acids

IM Hafez et al.

GENE THERAPY (2001)