Advances in the delivery of RNA therapeutics: from concept to clinical reality

Therapeutic reduction of ataxin-2 extends lifespan and reduces pathology in TDP-43 mice

(2017) Lindsay A. Becker et al.   NATURE

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

(2017) Norbert Pardi et al.   NATURE

Antisense oligonucleotide therapy for spinocerebellar ataxia type 2

(2017) Daniel R. Scoles et al.   NATURE

Chemical and structural effects of base modifications in messenger RNA

(2017) Emily M. Harcourt et al.   NATURE

Spinal muscular atrophy approval boosts antisense drugs

(2017) Elie Dolgin   NATURE BIOTECHNOLOGY

The 'anti-hype' vaccine

(2017) Laura DeFrancesco   NATURE BIOTECHNOLOGY

Cellular uptake and trafficking of antisense oligonucleotides

(2017) Stanley T Crooke et al.   NATURE BIOTECHNOLOGY

Overcoming cellular barriers for RNA therapeutics

(2017) Steven F Dowdy   NATURE BIOTECHNOLOGY

N1-methyl-pseudouridine in mRNA enhances translation through eIF2α-dependent and independent mechanisms by increasing ribosome density

(2017) Yuri V. Svitkin et al.   NUCLEIC ACIDS RESEARCH

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

Therapeutic genome engineering via CRISPR-Cas systems

(2017) Ana M. Moreno et al.   Wiley Interdisciplinary Reviews-Systems Biology and Medicine

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

(2017) Norbert Pardi et al.   Nature Communications

Genome Editing for the Study of Cardiovascular Diseases

(2017) Alexandra C. Chadwick et al.   Current Cardiology Reports

PGMA-Based Star-Like Polycations with Plentiful Hydroxyl Groups Act as Highly Efficient miRNA Delivery Nanovectors for Effective Applications in Heart Diseases

(2016) Rui-Quan Li et al.   ADVANCED MATERIALS

Bioinspired Alkenyl Amino Alcohol Ionizable Lipid Materials for Highly Potent In Vivo mRNA Delivery

(2016) Owen S. Fenton et al.   ADVANCED MATERIALS

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

Polymer-Lipid Nanoparticles for Systemic Delivery of mRNA to the Lungs

(2016) James C. Kaczmarek et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

Effects of Chemically Modified Messenger RNA on Protein Expression

(2016) Bin Li et al.   BIOCONJUGATE CHEMISTRY

Efficacy and immunogenicity of unmodified and pseudouridine-modified mRNA delivered systemically with lipid nanoparticles in vivo

(2016) Kevin J. Kauffman et al.   BIOMATERIALS

Exon Skipping Therapy

(2016) Courtney S. Young et al.   CELL

Recent innovations in mRNA vaccines

(2016) Jeffrey B Ulmer et al.   CURRENT OPINION IN IMMUNOLOGY

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

(2016) F DeRosa et al.   GENE THERAPY

CRISPR-Cas9 gene editing: Delivery aspects and therapeutic potential

(2016) Erik Oude Blenke et al.   JOURNAL OF CONTROLLED RELEASE

Methods for Optimizing CRISPR-Cas9 Genome Editing Specificity

(2016) Josh Tycko et al.   MOLECULAR CELL

Lipid Nanoparticle Assisted mRNA Delivery for Potent Cancer Immunotherapy

(2016) Matthias A. Oberli et al.   NANO LETTERS

In vivo genome editing via CRISPR/Cas9 mediated homology-independent targeted integration

(2016) Keiichiro Suzuki et al.   NATURE

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

(2016) Lena M. Kranz et al.   NATURE

In vitro and ex vivo strategies for intracellular delivery

(2016) Martin P. Stewart et al.   NATURE

High-fidelity CRISPR–Cas9 nucleases with no detectable genome-wide off-target effects

(2016) Benjamin P. Kleinstiver et al.   NATURE

Alnylam terminates revusiran program, stock plunges

(2016) Ken Garber   NATURE BIOTECHNOLOGY

Translation efficiency of mRNAs is increased by antisense oligonucleotides targeting upstream open reading frames

(2016) Xue-hai Liang et al.   NATURE BIOTECHNOLOGY

Enhancing homology-directed genome editing by catalytically active and inactive CRISPR-Cas9 using asymmetric donor DNA

(2016) Christopher D Richardson et al.   NATURE BIOTECHNOLOGY

Multiplex gene editing by CRISPR–Cpf1 using a single crRNA array

(2016) Bernd Zetsche et al.   NATURE BIOTECHNOLOGY

A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice

(2016) Yang Yang et al.   NATURE BIOTECHNOLOGY

Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo

(2016) Hao Yin et al.   NATURE BIOTECHNOLOGY

A multifunctional AAV–CRISPR–Cas9 and its host response

(2016) Wei Leong Chew et al.   NATURE METHODS

Editing the epigenome: technologies for programmable transcription and epigenetic modulation

(2016) Pratiksha I Thakore et al.   NATURE METHODS

Cornerstones of CRISPR–Cas in drug discovery and therapy

(2016) Christof Fellmann et al.   NATURE REVIEWS DRUG DISCOVERY

Coming of age: ten years of next-generation sequencing technologies

(2016) Sara Goodwin et al.   NATURE REVIEWS GENETICS

Dendrimer-RNA nanoparticles generate protective immunity against lethal Ebola, H1N1 influenza, andToxoplasma gondiichallenges with a single dose

(2016) Jasdave S. Chahal et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Efficient delivery of genome-editing proteins using bioreducible lipid nanoparticles

(2016) Ming Wang et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

From the RNA world to the clinic

(2016) B. A. Sullenger et al.   SCIENCE

RS-1 enhances CRISPR/Cas9- and TALEN-mediated knock-in efficiency

(2016) Jun Song et al.   Nature Communications

Surface charge tunability as a powerful strategy to control electrostatic interaction for high efficiency silencing, using tailored oligopeptide-modified poly(beta-amino ester)s (PBAEs)

(2015) Pere Dosta et al.   Acta Biomaterialia

Codon Optimality Is a Major Determinant of mRNA Stability

(2015) Vladimir Presnyak et al.   CELL

Cpf1 Is a Single RNA-Guided Endonuclease of a Class 2 CRISPR-Cas System

(2015) Bernd Zetsche et al.   CELL

Inhibition of nonhomologous end joining to increase the specificity of CRISPR/Cas9 genome editing

(2015) Supriya V. Vartak et al.   FEBS Journal

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

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

(2015) Andreas Thess et al.   MOLECULAR THERAPY

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

Mammalian synthetic circuits with RNA binding proteins for RNA-only delivery

(2015) Liliana Wroblewska et al.   NATURE BIOTECHNOLOGY

Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cells

(2015) Ayal Hendel et al.   NATURE BIOTECHNOLOGY

Orthogonal gene knockout and activation with a catalytically active Cas9 nuclease

(2015) James E Dahlman et al.   NATURE BIOTECHNOLOGY

Therapeutic genome editing: prospects and challenges

(2015) David Benjamin Turitz Cox et al.   NATURE MEDICINE

CRISPRscan: designing highly efficient sgRNAs for CRISPR-Cas9 targeting in vivo

(2015) Miguel A Moreno-Mateos et al.   NATURE METHODS

Knocking down disease: a progress report on siRNA therapeutics

(2015) Anders Wittrup et al.   NATURE REVIEWS GENETICS

Efficacy and safety of patisiran for familial amyloidotic polyneuropathy: a phase II multi-dose study

(2015) Ole B Suhr et al.   Orphanet Journal of Rare Diseases

Rationally engineered Cas9 nucleases with improved specificity

(2015) I. M. Slaymaker et al.   SCIENCE

Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy

(2015) C. Long et al.   SCIENCE

In vivo gene editing in dystrophic mouse muscle and muscle stem cells

(2015) M. Tabebordbar et al.   SCIENCE

In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy

(2015) C. E. Nelson et al.   SCIENCE

RNAi therapy targeting KRAS in combination with chemotherapy for locally advanced pancreatic cancer patients

(2015) Talia Golan et al.   Oncotarget

Pharmacological inhibition of DNA-PK stimulates Cas9-mediated genome editing

(2015) Francis Robert et al.   Genome Medicine

Abstract CT327: Multicenter phase I study of MRX34, a first-in-class microRNA miR-34 mimic liposomal injection

(2015) Muhammad Shaalan Beg et al.   CANCER RESEARCH

The complexity of miRNA-mediated repression

(2014) A Wilczynska et al.   CELL DEATH AND DIFFERENTIATION

Permanent Alteration of PCSK9 With In Vivo CRISPR-Cas9 Genome Editing

(2014) Qiurong Ding et al.   CIRCULATION RESEARCH

Nucleic acid vaccines: prospects for non-viral delivery of mRNA vaccines

(2014) Raquel P Deering et al.   Expert Opinion on Drug Delivery

VCAM-1 specific PEGylated SAINT-based lipoplexes deliver siRNA to activated endothelium in vivo but do not attenuate target gene expression

(2014) Niek G.J. Leus et al.   INTERNATIONAL JOURNAL OF PHARMACEUTICS

Multivalent N-Acetylgalactosamine-Conjugated siRNA Localizes in Hepatocytes and Elicits Robust RNAi-Mediated Gene Silencing

(2014) Jayaprakash K. Nair et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Cationic lipid-mediated delivery of proteins enables efficient protein-based genome editing in vitro and in vivo

(2014) John A Zuris et al.   NATURE BIOTECHNOLOGY

Improving CRISPR-Cas nuclease specificity using truncated guide RNAs

(2014) Yanfang Fu et al.   NATURE BIOTECHNOLOGY

Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype

(2014) Hao Yin et al.   NATURE BIOTECHNOLOGY

In vivo endothelial siRNA delivery using polymeric nanoparticles with low molecular weight

(2014) James E. Dahlman et al.   Nature Nanotechnology

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

Engineering adeno-associated viruses for clinical gene therapy

(2014) Melissa A. Kotterman et al.   NATURE REVIEWS GENETICS

Non-viral vectors for gene-based therapy

(2014) Hao Yin et al.   NATURE REVIEWS GENETICS

RNA-directed gene editing specifically eradicates latent and prevents new HIV-1 infection

(2014) W. Hu et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Lipopeptide nanoparticles for potent and selective siRNA delivery in rodents and nonhuman primates

(2014) Y. Dong et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

The new frontier of genome engineering with CRISPR-Cas9

(2014) J. A. Doudna et al.   SCIENCE

Genetics and genomics of Parkinson’s disease

(2014) Michelle K Lin et al.   Genome Medicine

Mechanism of Polyplex- and Lipoplex-Mediated Delivery of Nucleic Acids: Real-Time Visualization of Transient Membrane Destabilization without Endosomal Lysis

(2013) Zia ur Rehman et al.   ACS Nano

Eteplirsen for the treatment of Duchenne muscular dystrophy

(2013) Jerry R. Mendell et al.   ANNALS OF NEUROLOGY

A bioreducible linear poly(β-amino ester) for siRNA delivery

(2013) Kristen L. Kozielski et al.   CHEMICAL COMMUNICATIONS

Progress in microRNA delivery

(2013) Yu Zhang et al.   JOURNAL OF CONTROLLED RELEASE

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

(2013) Lior Zangi et al.   NATURE BIOTECHNOLOGY

Delivery materials for siRNA therapeutics

(2013) Rosemary Kanasty et al.   NATURE MATERIALS

Safety and Efficacy of RNAi Therapy for Transthyretin Amyloidosis

(2013) Teresa Coelho et al.   NEW ENGLAND JOURNAL OF MEDICINE

Multiplex Genome Engineering Using CRISPR/Cas Systems

(2013) L. Cong et al.   SCIENCE

Cystamine-terminated poly(beta-amino ester)s for siRNA delivery to human mesenchymal stem cells and enhancement of osteogenic differentiation

(2012) Stephany Y. Tzeng et al.   BIOMATERIALS

Delivering the promise of miRNA cancer therapeutics

(2012) Diane M. Pereira et al.   DRUG DISCOVERY TODAY

Rapid Discovery of Potent siRNA-Containing Lipid Nanoparticles Enabled by Controlled Microfluidic Formulation

(2012) Delai Chen et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

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

(2012) Benjamin Petsch et al.   NATURE BIOTECHNOLOGY

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

(2012) M. Jinek et al.   SCIENCE

A phase I/IIa immunotherapy trial of HIV-1-infected patients with Tat, Rev and Nef expressing dendritic cells followed by treatment interruption

(2011) Sabine D. Allard et al.   CLINICAL IMMUNOLOGY

Systemic delivery of therapeutic small interfering RNA using a pH-triggered amphiphilic poly-l-lysine nanocarrier to suppress prostate cancer growth in mice

(2011) Jianfeng Guo et al.   EUROPEAN JOURNAL OF PHARMACEUTICAL SCIENCES

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

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

Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges

(2011) Federico Mingozzi et al.   NATURE REVIEWS GENETICS

The Streptococcus thermophilus CRISPR/Cas system provides immunity in Escherichia coli

(2011) Rimantas Sapranauskas et al.   NUCLEIC ACIDS RESEARCH

Exploring the Genomes of Cancer Cells: Progress and Promise

(2011) M. R. Stratton   SCIENCE

Endocytosis of nanomedicines

(2010) Gaurav Sahay et al.   JOURNAL OF CONTROLLED RELEASE

mRNA as gene therapeutic: How to control protein expression

(2010) Geertrui Tavernier et al.   JOURNAL OF CONTROLLED RELEASE

Mipomersen, an apolipoprotein B synthesis inhibitor, for lowering of LDL cholesterol concentrations in patients with homozygous familial hypercholesterolaemia: a randomised, double-blind, placebo-controlled trial

(2010) Frederick J Raal et al.   LANCET

Lipid-like materials for low-dose, in vivo gene silencing

(2010) K. T. Love et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Functional genomics and rheumatoid arthritis: where have we been and where should we go?

(2010) James N Jarvis et al.   Genome Medicine

The RNA-induced Silencing Complex: A Versatile Gene-silencing Machine

(2009) Ashley J. Pratt et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Missing pieces in understanding the intracellular trafficking of polycation/DNA complexes

(2009) You-Yeon Won et al.   JOURNAL OF CONTROLLED RELEASE

Lipid-based nanotherapeutics for siRNA delivery

(2009) A. Schroeder et al.   JOURNAL OF INTERNAL MEDICINE

Development of Lipidoid–siRNA Formulations for Systemic Delivery to the Liver

(2009) Akin Akinc et al.   MOLECULAR THERAPY

A large-scale chemical modification screen identifies design rules to generate siRNAs with high activity, high stability and low toxicity

(2009) Jesper B. Bramsen et al.   NUCLEIC ACIDS RESEARCH

Systems medicine: the future of medical genomics and healthcare

(2009) Charles Auffray et al.   Genome Medicine

Antisense Oligonucleotide Directed to Human Apolipoprotein B-100 Reduces Lipoprotein(a) Levels and Oxidized Phospholipids on Human Apolipoprotein B-100 Particles in Lipoprotein(a) Transgenic Mice

(2008) Esther Merki et al.   CIRCULATION

Overcoming the Innate Immune Response to Small Interfering RNA

(2008) Adam Judge et al.   HUMAN GENE THERAPY

Antibody-Mediated Targeting of siRNA via the Human Insulin Receptor Using Avidin−Biotin Technology

(2008) Chun-Fang Xia et al.   MOLECULAR PHARMACEUTICS

Efficient In Vivo Delivery of siRNA to the Liver by Conjugation of α-Tocopherol

(2008) Kazutaka Nishina et al.   MOLECULAR THERAPY

Sequence- and target-independent angiogenesis suppression by siRNA via TLR3

(2008) Mark E. Kleinman et al.   NATURE

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

(2008) Akin Akinc et al.   NATURE BIOTECHNOLOGY

Thirty years of Alzheimer's disease genetics: the implications of systematic meta-analyses

(2008) Lars Bertram et al.   NATURE REVIEWS NEUROSCIENCE

Chemical Modification of siRNAs for In Vivo Use

(2008) Mark A. Behlke   OLIGONUCLEOTIDES