Non-liver mRNA Delivery

Species-dependent in vivo mRNA delivery and cellular responses to nanoparticles

(2022) Marine Z. C. Hatit et al.   Nature Nanotechnology

A noninflammatory mRNA vaccine for treatment of experimental autoimmune encephalomyelitis

(2021) Christina Krienke et al.   SCIENCE

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

(2021) Emmeline L. Blanchard et al.   NATURE BIOTECHNOLOGY

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

(2021) Shuai Liu et al.   NATURE MATERIALS

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

(2021) Min Qiu et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Enhanced target cell specificity and uptake of lipid nanoparticles using RNA aptamers and peptides

(2021) Roslyn M Ray et al.   Beilstein Journal of Organic Chemistry

Delivery routes matter: Safety and efficacy of intratumoral immunotherapy

(2021) Emily De Lombaerde et al.   BIOCHIMICA ET BIOPHYSICA ACTA-REVIEWS ON CANCER

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

(2021) Kiran Musunuru et al.   NATURE

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

(2021) Tanja Rothgangl et al.   NATURE BIOTECHNOLOGY

Secreted Expression of mRNA‐Encoded Truncated ACE2 Variants for SARS‐CoV‐2 via Lipid‐Like Nanoassemblies

(2021) Min Li et al.   ADVANCED MATERIALS

CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis

(2021) Julian D. Gillmore et al.   NEW ENGLAND JOURNAL OF MEDICINE

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

(2021) Michael Segel et al.   SCIENCE

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

(2021) Dániel Szőke et al.   Nature Communications

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

Engineering Caveolae-Targeted Lipid Nanoparticles To Deliver mRNA to the Lungs

(2020) Qing Li et al.   ACS Chemical Biology

A Nanoprimer To Improve the Systemic Delivery of siRNA and mRNA

(2020) Nell R. M. Saunders et al.   NANO LETTERS

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

(2020) Qiang Cheng et al.   Nature Nanotechnology

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

(2020) Lisa A. Jackson et al.   NEW ENGLAND JOURNAL OF MEDICINE

Distinct conformational states of SARS-CoV-2 spike protein

(2020) Yongfei Cai et al.   SCIENCE

Systemic nanoparticle delivery of CRISPR-Cas9 ribonucleoproteins for effective tissue specific genome editing

(2020) Tuo Wei et al.   Nature Communications

Increased PIP3 activity blocks nanoparticle mRNA delivery

(2020) Kalina Paunovska et al.   Science Advances

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

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

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

(2020) Chunxi Zeng et al.   ADVANCED MATERIALS

Synthetic in vitro transcribed lncRNAs (SINEUPs) with chemical modifications enhance target mRNA translation

(2020) Naoko Toki et al.   FEBS LETTERS

Treating Cystic Fibrosis with mRNA and CRISPR

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

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

(2020) Xinfu Zhang et al.   Science Advances

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

(2020) Lindsey R. Baden 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

Lipid nanoparticles-encapsulated brain-derived neurotrophic factor mRNA delivered through the round window niche in the cochleae of guinea pigs

(2020) Toru Miwa et al.   EXPERIMENTAL BRAIN RESEARCH

Inhaled Nanoformulated mRNA Polyplexes for Protein Production in Lung Epithelium

(2019) Asha Kumari Patel et al.   ADVANCED MATERIALS

Self-assembled peptide–poloxamine nanoparticles enable in vitro and in vivo genome restoration for cystic fibrosis

(2019) Shan Guan et al.   Nature Nanotechnology

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

(2019) Kalina Paunovska et al.   ADVANCED MATERIALS

Elimination Pathways of Nanoparticles

(2019) Wilson Poon et al.   ACS Nano

Visualization of early events in mRNA vaccine delivery in non-human primates via PET–CT and near-infrared imaging

(2019) Kevin E. Lindsay et al.   Nature Biomedical Engineering

Fast and Efficient CRISPR/Cas9 Genome Editing In Vivo Enabled by Bioreducible Lipid and Messenger RNA Nanoparticles

(2019) Ji Liu et al.   ADVANCED MATERIALS

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

(2019) Siddharth Patel et al.   JOURNAL OF CONTROLLED RELEASE

Constrained Nanoparticles Deliver siRNA and sgRNA to T Cells In Vivo without Targeting Ligands

(2019) Melissa P. Lokugamage et al.   ADVANCED MATERIALS

Non-viral delivery of chemically modified mRNA to the retina: Subretinal versus intravitreal administration

(2019) Joke Devoldere et al.   JOURNAL OF CONTROLLED RELEASE

Mild Innate Immune Activation Overrides Efficient Nanoparticle‐Mediated RNA Delivery

(2019) Melissa P. Lokugamage et al.   ADVANCED MATERIALS

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

(2018) Kalina Paunovska et al.   ACS Nano

Modified ARCA analogs providing enhanced translational properties of capped mRNAs

(2018) Ilona Kocmik et al.   CELL CYCLE

5′-Phosphorothiolate Dinucleotide Cap Analogues: Reagents for Messenger RNA Modification and Potent Small-Molecular Inhibitors of Decapping Enzymes

(2018) Blazej A. Wojtczak et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

In Vivo Introduction of mRNA Encapsulated in Lipid Nanoparticles to Brain Neuronal Cells and Astrocytes via Intracerebroventricular Administration

(2018) Hiroki Tanaka et al.   MOLECULAR PHARMACEUTICS

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

(2018) Ema Robinson et al.   MOLECULAR THERAPY

A Direct Comparison of in Vitro and in Vivo Nucleic Acid Delivery Mediated by Hundreds of Nanoparticles Reveals a Weak Correlation

(2018) Kalina Paunovska et al.   NANO LETTERS

Lipid Nanoparticle Formulations for Enhanced Co-delivery of siRNA and mRNA

(2018) Rebecca L. Ball et al.   NANO LETTERS

Principles for targeting RNA with drug-like small molecules

(2018) Katherine Deigan Warner et al.   NATURE REVIEWS DRUG DISCOVERY

Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis

(2018) David Adams et al.   NEW ENGLAND JOURNAL OF MEDICINE

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

(2018) Jonathan D. Finn et al.   Cell Reports

Rapid, Single-Cell Analysis and Discovery of Vectored mRNA Transfection In Vivo with a loxP-Flanked tdTomato Reporter Mouse

(2018) Kevin J. Kauffman et al.   Molecular Therapy-Nucleic Acids

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

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

Modifying a Commonly Expressed Endocytic Receptor Retargets Nanoparticles in Vivo

(2018) Cory D. Sago et al.   NANO LETTERS

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

(2018) Ruchi Jain et al.   Nucleic Acid Therapeutics

Barcoding chemical modifications into nucleic acids improves drug stability in vivo

(2018) Cory D. Sago et al.   Journal of Materials Chemistry B

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

Nanoparticles That Deliver RNA to Bone Marrow Identified by in Vivo Directed Evolution

(2018) Cory D. Sago et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

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

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

Boosting Intracellular Delivery of Lipid Nanoparticle-Encapsulated mRNA

(2017) Siddharth Patel et al.   NANO LETTERS

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

(2017) Ugur Sahin et al.   NATURE

Barcoded nanoparticles for high throughput in vivo discovery of targeted therapeutics

(2017) James E. Dahlman et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

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

(2016) Jason B. Miller et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

Recognition of Endogenous Nucleic Acids by the Innate Immune System

(2016) Axel Roers et al.   IMMUNITY

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

(2016) Lena M. Kranz et al.   NATURE

Mechanism of hard-nanomaterial clearance by the liver

(2016) Kim M. Tsoi et al.   NATURE MATERIALS

Messenger RNA modifications: Form, distribution, and function

(2016) W. V. Gilbert et al.   SCIENCE

Theranostic barcoded nanoparticles for personalized cancer medicine

(2016) Zvi Yaari et al.   Nature Communications

Codon Optimality Is a Major Determinant of mRNA Stability

(2015) Vladimir Presnyak et al.   CELL

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

(2015) Norbert Pardi 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

Synthesis, properties, and biological activity of boranophosphate analogs of the mRNA cap: versatile tools for manipulation of therapeutically relevant cap-dependent processes

(2014) Joanna Kowalska et al.   NUCLEIC ACIDS RESEARCH

The biomolecular corona is retained during nanoparticle uptake and protects the cells from the damage induced by cationic nanoparticles until degraded in the lysosomes

(2013) Fengjuan Wang et al.   Nanomedicine-Nanotechnology Biology and Medicine

Transferrin-functionalized nanoparticles lose their targeting capabilities when a biomolecule corona adsorbs on the surface

(2013) Anna Salvati et al.   Nature Nanotechnology

mRNA secondary structure optimization using a correlated stem–loop prediction

(2013) Paulo Gaspar et al.   NUCLEIC ACIDS RESEARCH

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

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

Highly Efficient Reprogramming to Pluripotency and Directed Differentiation of Human Cells with Synthetic Modified mRNA

(2010) Luigi Warren et al.   Cell Stem Cell

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

(2010) Akin Akinc et al.   MOLECULAR THERAPY

Locked Nucleic Acid (LNA)-Modified Dinucleotide mRNA Cap Analogue: Synthesis, Enzymatic Incorporation, and Utilization

(2009) Anilkumar R. Kore et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

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

(2008) Katalin Karikó et al.   MOLECULAR THERAPY