mRNA in the Context of Protein Replacement Therapy

Next generation self-replicating RNA vectors for vaccines and immunotherapies

(2022) Parinaz Aliahmad et al.   CANCER GENE THERAPY

What Is Cystic Fibrosis?

(2022) Tori M. Endres et al.   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

Lyophilization provides long-term stability for a lipid nanoparticle-formulated, nucleoside-modified mRNA vaccine

(2022) Hiromi Muramatsu et al.   MOLECULAR THERAPY

Ribozyme Assays to Quantify the Capping Efficiency of In Vitro-Transcribed mRNA

(2022) Irena Vlatkovic et al.   Pharmaceutics

Quality by Design for enabling RNA platform production processes

(2022) Simon Daniel et al.   TRENDS IN BIOTECHNOLOGY

Development of an mRNA replacement therapy for phenylketonuria

(2022) Carlos G. Perez-Garcia et al.   Molecular Therapy-Nucleic Acids

Making COVID-19 mRNA vaccines accessible: challenges resolved

(2022) Sarfaraz K. Niazi   Expert Review of Vaccines

mRNA-based modalities for infectious disease management

(2022) Mengjie Zhang et al.   Nano Research

Spotlight on Givosiran as a Treatment Option for Adults with Acute Hepatic Porphyria: Design, Development, and Place in Therapy

(2022) Chaudry Nasir Majeed et al.   Drug Design Development and Therapy

Gram‐Scale mRNA Production Using a 250‐mL Single‐Use Bioreactor

(2022) Janja Skok et al.   CHEMIE INGENIEUR TECHNIK

Self-Amplifying RNA Approach for Protein Replacement Therapy

(2022) Dimitri Papukashvili et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Payload distribution and capacity of mRNA lipid nanoparticles

(2022) Sixuan Li et al.   Nature Communications

The landscape of mRNA nanomedicine

(2022) Xiangang Huang et al.   NATURE MEDICINE

Novel lipid combination for delivery of plasmid DNA to immune cells in the spleen

(2021) Seigo Kimura et al.   JOURNAL OF CONTROLLED RELEASE

BNT162b vaccines protect rhesus macaques from SARS-CoV-2

(2021) Annette B. Vogel et al.   NATURE

mRNA vaccines manufacturing: Challenges and bottlenecks

(2021) Sara Sousa Rosa et al.   VACCINE

The Limitless Future of RNA Therapeutics

(2021) Tulsi Ram Damase et al.   Frontiers in Bioengineering and Biotechnology

Strategies for controlling the innate immune activity of conventional and self-amplifying mRNA therapeutics: Getting the message across

(2021) An-Katrien Minnaert et al.   ADVANCED DRUG DELIVERY REVIEWS

An overview of rational design of mRNA-based therapeutics and vaccines

(2021) Kenneth K.W. To et al.   Expert Opinion on Drug Discovery

Modifications of mRNA vaccine structural elements for improving mRNA stability and translation efficiency

(2021) Sun Chang Kim et al.   Molecular & Cellular Toxicology

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

(2021) Ugur Sahin et al.   NATURE

Low-dose mRNA-1273 COVID-19 vaccine generates durable memory enhanced by cross-reactive T cells

(2021) Jose Mateus et al.   SCIENCE

Lipid nanoparticles for mRNA delivery

(2021) Xucheng Hou et al.   Nature Reviews Materials

Pharma 4.0 Continuous mRNA Drug Products Manufacturing

(2021) Andreas Ouranidis et al.   Pharmaceutics

Challenges of Storage and Stability of mRNA-Based COVID-19 Vaccines

(2021) Mohammad N. Uddin et al.   Vaccines

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

(2021) Manuel J. Carrasco et al.   Communications Biology

Roles of mRNA poly(A) tails in regulation of eukaryotic gene expression

(2021) Lori A. Passmore et al.   NATURE REVIEWS MOLECULAR CELL BIOLOGY

mRNA, a Revolution in Biomedicine

(2021) Bruno Baptista et al.   Pharmaceutics

mRNA Therapeutic Modalities Design, Formulation and Manufacturing under Pharma 4.0 Principles

(2021) Andreas Ouranidis et al.   Biomedicines

Nanomedicines to Deliver mRNA: State of the Art and Future Perspectives

(2020) Itziar Gómez-Aguado et al.   Nanomaterials

Deconvoluting Lipid Nanoparticle Structure for Messenger RNA Delivery

(2020) Yulia Eygeris et al.   NANO LETTERS

Characterization of spike glycoprotein of SARS-CoV-2 on virus entry and its immune cross-reactivity with SARS-CoV

(2020) Xiuyuan Ou et al.   Nature Communications

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

(2020) Kizzmekia S. Corbett et al.   NATURE

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

(2020) Pengxuan Zhao et al.   Bioactive Materials

Treating Cystic Fibrosis with mRNA and CRISPR

(2020) Alejandro Da Silva Sanchez et al.   HUMAN GENE THERAPY

Synthetic mRNA Encoding VEGF-A in Patients Undergoing Coronary Artery Bypass Grafting: Design of a Phase 2a Clinical Trial

(2020) Vesa Anttila et al.   Molecular Therapy-Methods & Clinical Development

Addressing the Cold Reality of mRNA Vaccine Stability

(2020) Daan J.A. Crommelin et al.   JOURNAL OF PHARMACEUTICAL SCIENCES

Efficacy and Safety of the mRNA-1273 SARS-CoV-2 Vaccine

(2020) Lindsey R. Baden et al.   NEW ENGLAND JOURNAL OF MEDICINE

RNA Modifications Modulate Activation of Innate Toll-Like Receptors

(2019) Isabel Freund et al.   Genes

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

Messenger RNA therapy for rare genetic metabolic diseases

(2019) Pedro Berraondo et al.   GUT

Phenylketonuria: Current Treatments and Future Developments

(2019) Uta Lichter-Konecki et al.   DRUGS

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

(2019) Kalina Paunovska et al.   ADVANCED MATERIALS

Inotersen: new promise for the treatment of hereditary transthyretin amyloidosis

(2019) Veena Mathew et al.   Drug Design Development and Therapy

Volanesorsen: First Global Approval

(2019) Julia Paik et al.   DRUGS

Correction of bleeding in experimental severe hemophilia A by systemic delivery of factor VIII-encoding mRNA

(2019) Jules Russick et al.   HAEMATOLOGICA

Messenger RNA therapy as an option for treating metabolic disorders

(2019) Randy J. Chandler   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

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

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

(2019) David M. Mauger et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Tailoring mRNA Vaccine to Balance Innate/Adaptive Immune Response

(2019) Sergio Linares-Fernández et al.   TRENDS IN MOLECULAR MEDICINE

GC content shapes mRNA storage and decay in human cells

(2019) Maïté Courel et al.   eLife

Analyzing 2000 in Vivo Drug Delivery Data Points Reveals Cholesterol Structure Impacts Nanoparticle Delivery

(2018) Kalina Paunovska et al.   ACS Nano

Poly A tail length analysis of in vitro transcribed mRNA by LC-MS

(2018) Michael Beverly et al.   ANALYTICAL AND BIOANALYTICAL CHEMISTRY

Lipid Polymer Hybrid Nanomaterials for mRNA Delivery

(2018) Weiyu Zhao et al.   Cellular and Molecular Bioengineering

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

(2018) Ema Robinson et al.   MOLECULAR THERAPY

Lipid Nanoparticles Enabling Gene Therapies: From Concepts to Clinical Utility

(2018) Jayesh A. Kulkarni et al.   Nucleic Acid Therapeutics

Biocompatible, Purified VEGF-A mRNA Improves Cardiac Function after Intracardiac Injection 1 Week Post-myocardial Infarction in Swine

(2018) Leif Carlsson et al.   Molecular Therapy-Methods & Clinical Development

What Are 3′ UTRs Doing?

(2018) Christine Mayr   Cold Spring Harbor Perspectives in Biology

Delivery of mRNA Therapeutics for the Treatment of Hepatic Diseases

(2018) Zeljka Trepotec et al.   MOLECULAR THERAPY

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-Based Protein Replacement Therapy for the Heart

(2018) Ajit Magadum et al.   MOLECULAR THERAPY

mRNA decapping: finding the right structures

(2018) Clément Charenton et al.   PHILOSOPHICAL TRANSACTIONS OF THE ROYAL SOCIETY B-BIOLOGICAL SCIENCES

Chemically modified hCFTR mRNAs recuperate lung function in a mouse model of cystic fibrosis

(2018) A. K. M. Ashiqul Haque et al.   Scientific Reports

Modified VEGF-A mRNA induces sustained multifaceted microvascular response and accelerates diabetic wound healing

(2018) Naidi Sun et al.   Scientific Reports

Safety and immunogenicity of a mRNA rabies vaccine in healthy adults: an open-label, non-randomised, prospective, first-in-human phase 1 clinical trial

(2017) Martin Alberer et al.   LANCET

Optimizing Cardiac Delivery of Modified mRNA

(2017) Nishat Sultana et al.   MOLECULAR THERAPY

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

Eteplirsen in the treatment of Duchenne muscular dystrophy

(2017) Kenji Rowel Lim et al.   Drug Design Development and Therapy

Surface De-PEGylation Controls Nanoparticle-Mediated siRNA Delivery In Vitro and In Vivo

(2017) Xi Zhu et al.   Theranostics

pEVL: A Linear Plasmid for Generating mRNA IVT Templates With Extended Encoded Poly(A) Sequences

(2016) Alexandra E Grier et al.   Molecular Therapy-Nucleic Acids

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

Genotype–Phenotype Correlations in Ornithine Transcarbamylase Deficiency: A Mutation Update

(2015) Ljubica Caldovic et al.   Journal of Genetics and Genomics

Arginase-1 deficiency

(2015) Yuan Yan Sin et al.   JOURNAL OF MOLECULAR MEDICINE-JMM

mRNA-based therapeutics — developing a new class of drugs

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

Determinants and implications of mRNA poly(A) tail size – Does this protein make my tail look big?

(2014) Aimee L. Jalkanen et al.   SEMINARS IN CELL & DEVELOPMENTAL BIOLOGY

Large-scale purification of pharmaceutical-grade plasmid DNA using tangential flow filtration and multi-step chromatography

(2013) Bo Sun et al.   JOURNAL OF BIOSCIENCE AND BIOENGINEERING

Modified mRNA directs the fate of heart progenitor cells and induces vascular regeneration after myocardial infarction

(2013) Lior Zangi et al.   NATURE BIOTECHNOLOGY

Efficient genome editing in zebrafish using a CRISPR-Cas system

(2013) Woong Y Hwang et al.   NATURE BIOTECHNOLOGY

Protein Replacement Therapies for Rare Diseases: A Breeze for Regulatory Approval?

(2013) J. A. Gorzelany et al.   Science Translational Medicine

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

(2012) Katalin Karikó et al.   MOLECULAR THERAPY

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

(2012) Benjamin Petsch et al.   NATURE BIOTECHNOLOGY

Nonviral delivery of self-amplifying RNA vaccines

(2012) A. J. Geall et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Developing mRNA-vaccine technologies

(2012) Thomas Schlake et al.   RNA Biology

Phenylalanine hydroxylase deficiency

(2011) John J. Mitchell et al.   GENETICS IN MEDICINE

Genome Analysis Reveals Interplay between 5′UTR Introns and Nuclear mRNA Export for Secretory and Mitochondrial Genes

(2011) Can Cenik et al.   PLoS Genetics

RNA Isolation from Mammalian Cells Using Porous Polymer Monoliths: An Approach for High-Throughput Automation

(2010) Anirban Chatterjee et al.   ANALYTICAL CHEMISTRY

Intranodal Vaccination with Naked Antigen-Encoding RNA Elicits Potent Prophylactic and Therapeutic Antitumoral Immunity

(2010) S. Kreiter et al.   CANCER RESEARCH

A Role for Codon Order in Translation Dynamics

(2010) Gina Cannarozzi et al.   CELL

Direct Injection of Protamine-protected mRNA: Results of a Phase 1/2 Vaccination Trial in Metastatic Melanoma Patients

(2009) Benjamin Weide et al.   JOURNAL OF IMMUNOTHERAPY

Optimizing the use of sapropterin (BH4) in the management of phenylketonuria

(2009) Nenad Blau et al.   MOLECULAR GENETICS AND METABOLISM

Defining tetrahydrobiopterin (BH4)-responsiveness in PKU

(2008) Nenad Blau   JOURNAL OF INHERITED METABOLIC DISEASE

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

(2008) Katalin Karikó et al.   MOLECULAR THERAPY