Non-Immunotherapy Application of LNP-mRNA: Maximizing Efficacy and Safety

An Update on Self-Amplifying mRNA Vaccine Development

(2021) Anna K. Blakney et al.   Vaccines

mRNA therapeutics in cancer immunotherapy

(2021) Jan D. Beck et al.   Molecular Cancer

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

(2021) Thai Thanh Hoang Thi et al.   Vaccines

A Combinatorial Library of Lipid Nanoparticles for RNA Delivery to Leukocytes

(2020) Srinivas Ramishetti et al.   ADVANCED MATERIALS

The identity and methylation status of the first transcribed nucleotide in eukaryotic mRNA 5′ cap modulates protein expression in living cells

(2020) Pawel J Sikorski et al.   NUCLEIC ACIDS RESEARCH

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

(2020) Oscar A. Marcos-Contreras et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Synthesis of low immunogenicity RNA with high-temperature in vitro transcription

(2020) Monica Z. Wu et al.   RNA

Naturally-occurring cholesterol analogues in lipid nanoparticles induce polymorphic shape and enhance intracellular delivery of mRNA

(2020) Siddharth Patel et al.   Nature Communications

Deconvoluting Lipid Nanoparticle Structure for Messenger RNA Delivery

(2020) Yulia Eygeris et al.   NANO LETTERS

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

(2020) Ahmad Karadagi et al.   Scientific Reports

Recent Advances in Understanding the Protein Corona of Nanoparticles and in the Formulation of “Stealthy” Nanomaterials

(2020) Riccardo Rampado et al.   Frontiers in Bioengineering and Biotechnology

Lipid nanoparticles for nucleic acid delivery: Current perspectives

(2020) Eleni Samaridou et al.   ADVANCED DRUG DELIVERY REVIEWS

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

(2020) Gergely Tibor Kozma et al.   ADVANCED DRUG DELIVERY REVIEWS

Recent advances in mRNA vaccine technology

(2020) Norbert Pardi et al.   CURRENT OPINION IN IMMUNOLOGY

SARS-CoV-2 mRNA vaccine design enabled by prototype pathogen preparedness

(2020) Kizzmekia S. Corbett et al.   NATURE

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

(2020) Zubao Gan et al.   Bioengineering & Translational Medicine

Self-amplifying RNA vaccines for infectious diseases

(2020) Kristie Bloom et al.   GENE THERAPY

PEG shedding-rate-dependent blood clearance of PEGylated lipid nanoparticles in mice: Faster PEG shedding attenuates anti-PEG IgM production

(2020) Takuya Suzuki et al.   INTERNATIONAL JOURNAL OF PHARMACEUTICS

The Ongoing Quest to Crack the Genetic Code for Protein Production

(2020) Thijs Nieuwkoop et al.   MOLECULAR CELL

COVID-19 vaccine BNT162b1 elicits human antibody and TH1 T cell responses

(2020) Ugur Sahin et al.   NATURE

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

(2020) Evan J. Anderson et al.   NEW ENGLAND JOURNAL OF MEDICINE

Cytokine Storm

(2020) David C. Fajgenbaum et al.   NEW ENGLAND JOURNAL OF MEDICINE

Safety and Efficacy of the BNT162b2 mRNA Covid-19 Vaccine

(2020) Fernando P. Polack et al.   NEW ENGLAND JOURNAL OF MEDICINE

Human NLRP1 is a sensor for double-stranded RNA

(2020) Stefan Bauernfried et al.   SCIENCE

Ciphers and Executioners: How 3′-Untranslated Regions Determine the Fate of Messenger RNAs

(2019) Vinay K. Mayya et al.   Frontiers in Genetics

Improved Efficacy in a Fabry Disease Model Using a Systemic mRNA Liver Depot System as Compared to Enzyme Replacement Therapy

(2019) Frank DeRosa et al.   MOLECULAR THERAPY

Delivering the Messenger: Advances in Technologies for Therapeutic mRNA Delivery

(2019) Piotr S. Kowalski et al.   MOLECULAR THERAPY

Systemic mRNA Therapy for the Treatment of Fabry Disease: Preclinical Studies in Wild-Type Mice, Fabry Mouse Model, and Wild-Type Non-human Primates

(2019) Xuling Zhu et al.   AMERICAN JOURNAL OF HUMAN GENETICS

mRNA Therapy Improves Metabolic and Behavioral Abnormalities in a Murine Model of Citrin Deficiency

(2019) Jingsong Cao et al.   MOLECULAR THERAPY

Toxicity and immunogenicity concerns related to PEGylated-micelle carrier systems: a review

(2019) Kouichi Shiraishi et al.   SCIENCE AND TECHNOLOGY OF ADVANCED MATERIALS

Optimization of Lipid Nanoparticles for Intramuscular Administration of mRNA Vaccines

(2019) Kimberly J. Hassett et al.   Molecular Therapy-Nucleic Acids

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

(2019) Markus Baiersdörfer et al.   Molecular Therapy-Nucleic Acids

Pseudo-Anaphylaxis to Polyethylene Glycol (PEG)-Coated Liposomes: Roles of Anti-PEG IgM and Complement Activation in a Porcine Model of Human Infusion Reactions

(2019) Gergely Tibor Kozma et al.   ACS Nano

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

(2019) Paul J. Sample et al.   NATURE BIOTECHNOLOGY

PEGylated liposomes: immunological responses

(2019) Marwa Mohamed et al.   SCIENCE AND TECHNOLOGY OF ADVANCED MATERIALS

ADAR1: “Editor-in-Chief” of Cytoplasmic Innate Immunity

(2019) Mart M. Lamers et al.   Frontiers in Immunology

Long-term efficacy and safety of mRNA therapy in two murine models of methylmalonic acidemia

(2019) Ding An et al.   EBioMedicine

A lipid-encapsulated mRNA encoding a potently neutralizing human monoclonal antibody protects against chikungunya infection

(2019) Nurgun Kose et al.   Science Immunology

Novel mRNA-Based Therapy Reduces Toxic Galactose Metabolites and Overcomes Galactose Sensitivity in a Mouse Model of Classic Galactosemia

(2019) Bijina Balakrishnan et al.   MOLECULAR THERAPY

FLAM-seq: full-length mRNA sequencing reveals principles of poly(A) tail length control

(2019) Ivano Legnini et al.   NATURE METHODS

Lipid nanoparticle-targeted mRNA therapy as a treatment for the inherited metabolic liver disorder arginase deficiency

(2019) Brian Truong et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

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

(2019) Marco Maugeri et al.   Nature Communications

mRNA as a Novel Treatment Strategy for Hereditary Spastic Paraplegia Type 5

(2019) Stefan Hauser et al.   Molecular Therapy-Methods & Clinical Development

Dexamethasone prodrugs as potent suppressors of the immunostimulatory effects of lipid nanoparticle formulations of nucleic acids

(2018) Sam Chen et al.   JOURNAL OF CONTROLLED RELEASE

G6PC mRNA Therapy Positively Regulates Fasting Blood Glucose and Decreases Liver Abnormalities in a Mouse Model of Glycogen Storage Disease 1a

(2018) Daniel S. Roseman et al.   MOLECULAR THERAPY

Lipid Nanoparticle-Delivered Chemically Modified mRNA Restores Chloride Secretion in Cystic Fibrosis

(2018) Ema Robinson et al.   MOLECULAR THERAPY

Targeted mRNA Therapy for Ornithine Transcarbamylase Deficiency

(2018) Mary G. Prieve et al.   MOLECULAR THERAPY

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

(2018) Staci Sabnis et al.   MOLECULAR THERAPY

mRNA vaccines — a new era in vaccinology

(2018) Norbert Pardi et al.   NATURE REVIEWS DRUG DISCOVERY

An origin of the immunogenicity of in vitro transcribed RNA

(2018) Xin Mu et al.   NUCLEIC ACIDS RESEARCH

Optimization of mRNA untranslated regions for improved expression of therapeutic mRNA

(2018) Kirtika H. Asrani et al.   RNA Biology

Arginase I mRNA therapy – A novel approach to rescue arginase 1 enzyme deficiency

(2018) Kirtika H. Asrani et al.   RNA Biology

mRNA treatment produces sustained expression of enzymatically active human ADAMTS13 in mice

(2018) Susan Liu-Chen et al.   Scientific Reports

Uridine Depletion and Chemical Modification Increase Cas9 mRNA Activity and Reduce Immunogenicity without HPLC Purification

(2018) Sriram Vaidyanathan et al.   Molecular Therapy-Nucleic Acids

Main trends of immune effects triggered by nanomedicines in preclinical studies

(2018) Blanka Halamoda-Kenzaoui et al.   International Journal of Nanomedicine

MicroRNAs Enable mRNA Therapeutics to Selectively Program Cancer Cells to Self-Destruct

(2018) Ruchi Jain et al.   Nucleic Acid Therapeutics

Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia

(2018) Ding An et al.   Cell Reports

PECAM-1 directed re-targeting of exogenous mRNA providing two orders of magnitude enhancement of vascular delivery and expression in lungs independent of apolipoprotein E-mediated uptake

(2018) Hamideh Parhiz et al.   JOURNAL OF CONTROLLED RELEASE

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

(2018) Alexandra G. Orlandini von Niessen et al.   MOLECULAR THERAPY

mRNA therapeutics deliver a hopeful message

(2018) Zifu Zhong et al.   Nano Today

Systemic messenger RNA as an etiological treatment for acute intermittent porphyria

(2018) Lei Jiang et al.   NATURE MEDICINE

Inosine induces context-dependent recoding and translational stalling

(2018) Konstantin Licht et al.   NUCLEIC ACIDS RESEARCH

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

(2018) Cory D. Sago et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Cell specific delivery of modified mRNA expressing therapeutic proteins to leukocytes

(2018) Nuphar Veiga et al.   Nature Communications

Preclinical and Clinical Demonstration of Immunogenicity by mRNA Vaccines against H10N8 and H7N9 Influenza Viruses

(2017) Kapil Bahl et al.   MOLECULAR THERAPY

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

(2017) Norbert Pardi et al.   NATURE

Elimination of large tumors in mice by mRNA-encoded bispecific antibodies

(2017) Christiane R Stadler et al.   NATURE MEDICINE

Systemic delivery of factor IX messenger RNA for protein replacement therapy

(2017) Suvasini Ramaswamy et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

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

(2017) Yazan M. Abbas et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Effects of engineered nanoparticles on the innate immune system

(2017) Yuanchang Liu et al.   SEMINARS IN IMMUNOLOGY

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

(2017) Maja Sedic et al.   VETERINARY PATHOLOGY

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

(2017) Norbert Pardi et al.   Nature Communications

MAVS activates TBK1 and IKKε through TRAFs in NEMO dependent and independent manner

(2017) Run Fang et al.   PLoS Pathogens

Ribonuclease L mediates the cell-lethal phenotype of double-stranded RNA editing enzyme ADAR1 deficiency in a human cell line

(2017) Yize Li et al.   eLife

Regulation of Macrophage Recognition through the Interplay of Nanoparticle Surface Functionality and Protein Corona

(2016) Krishnendu Saha et al.   ACS Nano

PEGylation as a strategy for improving nanoparticle-based drug and gene delivery

(2016) Jung Soo Suk et al.   ADVANCED DRUG DELIVERY REVIEWS

Preclinical evaluation of RNAi as a treatment for transthyretin-mediated amyloidosis

(2016) James S. Butler et al.   AMYLOID-JOURNAL OF PROTEIN FOLDING DISORDERS

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

(2016) Joke Devoldere et al.   DRUG DISCOVERY TODAY

Therapeutic efficacy in a hemophilia B model using a biosynthetic mRNA liver depot system

(2016) F DeRosa et al.   GENE THERAPY

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

(2016) Lena M. Kranz et al.   NATURE

mRNA capping: biological functions and applications

(2016) Anand Ramanathan et al.   NUCLEIC ACIDS RESEARCH

Structural basis for m7G recognition and 2′-O-methyl discrimination in capped RNAs by the innate immune receptor RIG-I

(2016) Swapnil C. Devarkar et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Translational control by 5'-untranslated regions of eukaryotic mRNAs

(2016) A. G. Hinnebusch et al.   SCIENCE

Intrathecal delivery of frataxin mRNA encapsulated in lipid nanoparticles to dorsal root ganglia as a potential therapeutic for Friedreich’s ataxia

(2016) Joseph F. Nabhan et al.   Scientific Reports

New insights into decapping enzymes and selective mRNA decay

(2016) Ewa Grudzien-Nogalska et al.   Wiley Interdisciplinary Reviews-RNA

Systemic Gene Silencing in Primary T Lymphocytes Using Targeted Lipid Nanoparticles

(2015) Srinivas Ramishetti et al.   ACS Nano

Critical residues involved in Toll-like receptor 4 activation by cationic lipid nanocarriers are not located at the lipopolysaccharide-binding interface

(2015) Caroline Lonez et al.   CELLULAR AND MOLECULAR LIFE SCIENCES

A Conserved Histidine in the RNA Sensor RIG-I Controls Immune Tolerance to N1-2′O-Methylated Self RNA

(2015) Christine Schuberth-Wagner et al.   IMMUNITY

N 1 -methylpseudouridine-incorporated mRNA outperforms pseudouridine-incorporated mRNA by providing enhanced protein expression and reduced immunogenicity in mammalian cell lines and mice

(2015) Oliwia Andries et al.   JOURNAL OF CONTROLLED RELEASE

Optimization of Lipid Nanoparticle Formulations for mRNA Delivery in Vivo with Fractional Factorial and Definitive Screening Designs

(2015) Kevin J. Kauffman et al.   NANO LETTERS

Cleavage of GSDMD by inflammatory caspases determines pyroptotic cell death

(2015) Jianjin Shi et al.   NATURE

A-to-I editing of coding and non-coding RNAs by ADARs

(2015) Kazuko Nishikura   NATURE REVIEWS MOLECULAR CELL BIOLOGY

RNA editing by ADAR1 prevents MDA5 sensing of endogenous dsRNA as nonself

(2015) B. J. Liddicoat et al.   SCIENCE

IFIT1: A dual sensor and effector molecule that detects non-2′-O methylated viral RNA and inhibits its translation

(2014) Michael S. Diamond   CYTOKINE & GROWTH FACTOR REVIEWS

TAIL-seq: Genome-wide Determination of Poly(A) Tail Length and 3′ End Modifications

(2014) Hyeshik Chang et al.   MOLECULAR CELL

Complement activation-related pseudoallergy: A stress reaction in blood triggered by nanomedicines and biologicals

(2014) Janos Szebeni   MOLECULAR IMMUNOLOGY

Antiviral immunity via RIG-I-mediated recognition of RNA bearing 5′-diphosphates

(2014) Delphine Goubau et al.   NATURE

Poly(A)-tail profiling reveals an embryonic switch in translational control

(2014) Alexander O. Subtelny et al.   NATURE

Overcoming the challenges in administering biopharmaceuticals: formulation and delivery strategies

(2014) Samir Mitragotri et al.   NATURE REVIEWS DRUG DISCOVERY

mRNA-based therapeutics — developing a new class of drugs

(2014) Ugur Sahin et al.   NATURE REVIEWS DRUG DISCOVERY

A critical analysis of codon optimization in human therapeutics

(2014) Vincent P. Mauro et al.   TRENDS IN MOLECULAR MEDICINE

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

(2014) Varun Kumar et al.   Molecular Therapy-Nucleic Acids

NOD-Like Receptors: Master Regulators of Inflammation and Cancer

(2014) Mansi Saxena et al.   Frontiers in Immunology

Cytokines as biomarkers of nanoparticle immunotoxicity

(2013) Mahmoud Elsabahy et al.   CHEMICAL SOCIETY REVIEWS

Innate immune response and programmed cell death following carrier-mediated delivery of unmodified mRNA to respiratory cells

(2013) Oliwia Andries et al.   JOURNAL OF CONTROLLED RELEASE

The accelerated blood clearance (ABC) phenomenon: Clinical challenge and approaches to manage

(2013) Amr S. Abu Lila et al.   JOURNAL OF CONTROLLED RELEASE

Cytokine Levels in the Serum of Healthy Subjects

(2013) Giulio Kleiner et al.   MEDIATORS OF INFLAMMATION

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

(2013) Martin A Maier et al.   MOLECULAR THERAPY

Cationic lipid nanocarriers activate Toll-like receptor 2 and NLRP3 inflammasome pathways

(2013) Caroline Lonez et al.   Nanomedicine-Nanotechnology Biology and Medicine

Nucleic Acid Delivery: The Missing Pieces of the Puzzle?

(2012) Juliane Nguyen et al.   ACCOUNTS OF CHEMICAL RESEARCH

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

(2011) Michael S D Kormann et al.   NATURE BIOTECHNOLOGY

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

(2011) Roland Züst et al.   NATURE IMMUNOLOGY

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

(2011) Katalin Karikó et al.   NUCLEIC ACIDS RESEARCH

The systemic toxicity of positively charged lipid nanoparticles and the role of Toll-like receptor 4 in immune activation

(2010) Ranit Kedmi et al.   BIOMATERIALS

Phosphorothioate cap analogs increase stability and translational efficiency of RNA vaccines in immature dendritic cells and induce superior immune responses in vivo

(2010) A N Kuhn et al.   GENE THERAPY

Variable Antibody-dependent Activation of Complement by Functionalized Phospholipid Nanoparticle Surfaces

(2010) Christine T. N. Pham et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

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

(2010) Akin Akinc et al.   MOLECULAR THERAPY

Rational design of cationic lipids for siRNA delivery

(2010) Sean C Semple et al.   NATURE BIOTECHNOLOGY

Structural and functional insights into 5′-ppp RNA pattern recognition by the innate immune receptor RIG-I

(2010) Yanli Wang et al.   NATURE STRUCTURAL & MOLECULAR BIOLOGY

Activation of MDA5 Requires Higher-Order RNA Structures Generated during Virus Infection

(2009) A. Pichlmair et al.   JOURNAL OF VIROLOGY

Evaluation of Efficacy, Biodistribution, and Inflammation for a Potent siRNA Nanoparticle: Effect of Dexamethasone Co-treatment

(2009) Marc T Abrams et al.   MOLECULAR THERAPY

Knocking down barriers: advances in siRNA delivery

(2009) Kathryn A. Whitehead et al.   NATURE REVIEWS DRUG DISCOVERY

5'-triphosphate RNA requires base-paired structures to activate antiviral signaling via RIG-I

(2009) A. Schmidt et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

The size of endothelial fenestrae in human liver sinusoids: implications for hepatocyte-directed gene transfer

(2008) E Wisse et al.   GENE THERAPY

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

(2008) Katalin Karikó et al.   MOLECULAR THERAPY

ADAR1 is essential for the maintenance of hematopoiesis and suppression of interferon signaling

(2008) Jochen C Hartner et al.   NATURE IMMUNOLOGY

Synthesis and characterization of mRNA cap analogs containing phosphorothioate substitutions that bind tightly to eIF4E and are resistant to the decapping pyrophosphatase DcpS

(2008) J. Kowalska et al.   RNA