The current landscape of nucleic acid therapeutics

In vivo cytidine base editing of hepatocytes without detectable off-target mutations in RNA and DNA

(2021) Lukas Villiger et al.   Nature Biomedical Engineering

Density Matching Multi-wavelength Analytical Ultracentrifugation to Measure Drug Loading of Lipid Nanoparticle Formulations

(2021) Amy Henrickson et al.   ACS Nano

Anti-PfGARP activates programmed cell death of parasites and reduces severe malaria

(2020) Dipak K. Raj et al.   NATURE

Directed evolution of adenine base editors with increased activity and therapeutic application

(2020) Nicole M. Gaudelli et al.   NATURE BIOTECHNOLOGY

The COVID-19 vaccine development landscape

(2020) Tung Thanh Le et al.   NATURE REVIEWS DRUG DISCOVERY

Developing Covid-19 Vaccines at Pandemic Speed

(2020) Nicole Lurie et al.   NEW ENGLAND JOURNAL OF MEDICINE

Chimeric siRNAs with chemically modified pentofuranose and hexopyranose nucleotides: altritol-nucleotide (ANA) containing GalNAc–siRNA conjugates: in vitro and in vivo RNAi activity and resistance to 5′-exonuclease

(2020) Pawan Kumar et al.   NUCLEIC ACIDS RESEARCH

Generation and validation of structurally defined antibody–siRNA conjugates

(2020) Alex R Nanna et al.   NUCLEIC ACIDS RESEARCH

Oligonucleotides and the COVID-19 Pandemic: A Perspective

(2020) John J. Rossi et al.   Nucleic Acid Therapeutics

Lipid nanoparticle technology for therapeutic gene regulation in the liver

(2020) Dominik Witzigmann et al.   ADVANCED DRUG DELIVERY REVIEWS

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

(2020) Kizzmekia S. Corbett et al.   NATURE

Phase 1/2 study of COVID-19 RNA vaccine BNT162b1 in adults

(2020) Mark J. Mulligan et al.   NATURE

Genome editing with CRISPR–Cas nucleases, base editors, transposases and prime editors

(2020) Andrew V. Anzalone et al.   NATURE BIOTECHNOLOGY

Advances in oligonucleotide drug delivery

(2020) Thomas C. Roberts et al.   NATURE REVIEWS DRUG DISCOVERY

Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria

(2020) Manisha Balwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

Self-amplifying RNA SARS-CoV-2 lipid nanoparticle vaccine candidate induces high neutralizing antibody titers in mice

(2020) Paul F. McKay et al.   Nature Communications

Investigating the pharmacodynamic durability of GalNAc–siRNA conjugates

(2020) Christopher R Brown et al.   NUCLEIC ACIDS RESEARCH

Synthesis, chirality-dependent conformational and biological properties of siRNAs containing 5′-(R)- and 5′-(S)-C-methyl-guanosine

(2020) Atsushi Mikami et al.   NUCLEIC ACIDS RESEARCH

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

Bioconjugated oligonucleotides: recent developments and therapeutic applications

(2019) sebastien benizri et al.   BIOCONJUGATE CHEMISTRY

Phase 1 Trial of an RNA Interference Therapy for Acute Intermittent Porphyria

(2019) Eliane Sardh et al.   NEW ENGLAND JOURNAL OF MEDICINE

Adeno-associated virus vector as a platform for gene therapy delivery

(2019) Dan Wang et al.   NATURE REVIEWS DRUG DISCOVERY

Fatty acid conjugation enhances potency of antisense oligonucleotides in muscle

(2019) Thazha P Prakash et al.   NUCLEIC ACIDS RESEARCH

The Onpattro story and the clinical translation of nanomedicines containing nucleic acid-based drugs

(2019) Akin Akinc et al.   Nature Nanotechnology

On the Formation and Morphology of Lipid Nanoparticles Containing Ionizable Cationic Lipids and siRNA

(2018) Jayesh A. Kulkarni et al.   ACS Nano

Evaluation of GalNAc-siRNA Conjugate Activity in Pre-clinical Animal Models with Reduced Asialoglycoprotein Receptor Expression

(2018) Jennifer L.S. Willoughby et al.   MOLECULAR THERAPY

Advanced siRNA Designs Further Improve In Vivo Performance of GalNAc-siRNA Conjugates

(2018) Donald J. Foster et al.   MOLECULAR THERAPY

mRNA vaccines — a new era in vaccinology

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

Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis

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

Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis

(2018) Merrill D. Benson et al.   NEW ENGLAND JOURNAL OF MEDICINE

Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy

(2018) Eugenio Mercuri et al.   NEW ENGLAND JOURNAL OF MEDICINE

Personalized vaccines for cancer immunotherapy

(2018) Ugur Sahin et al.   SCIENCE

CAR T cell immunotherapy for human cancer

(2018) Carl H. June et al.   SCIENCE

Gene therapy comes of age

(2018) Cynthia E. Dunbar et al.   SCIENCE

Cholesterol–GalNAc Dual Conjugation Strategy for Reducing Renal Distribution of Antisense Oligonucleotides

(2018) Fumito Wada et al.   Nucleic Acid Therapeutics

Lipid Nanoparticles Enabling Gene Therapies: From Concepts to Clinical Utility

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

GalNAc-siRNA Conjugates: Leading the Way for Delivery of RNAi Therapeutics

(2018) Aaron D. Springer et al.   Nucleic Acid Therapeutics

Nucleoside-modified mRNA immunization elicits influenza virus hemagglutinin stalk-specific antibodies

(2018) Norbert Pardi et al.   Nature Communications

Sangamo's landmark genome editing trial gets mixed reception

(2018) Cormac Sheridan   NATURE BIOTECHNOLOGY

Treatment of a metabolic liver disease by in vivo genome base editing in adult mice

(2018) Lukas Villiger et al.   NATURE MEDICINE

Diverse lipid conjugates for functional extra-hepatic siRNA delivery in vivo

(2018) Annabelle Biscans et al.   NUCLEIC ACIDS RESEARCH

The Effectiveness and Value of Patisiran and Inotersen for Hereditary Transthyretin Amyloidosis

(2018) Kristin Mickle et al.   Journal of Managed Care & Specialty Pharmacy

Optimization of design and production strategies for novel adeno-associated viral display peptide libraries

(2017) J Körbelin et al.   GENE THERAPY

Chirality Dependent Potency Enhancement and Structural Impact of Glycol Nucleic Acid Modification on siRNA

(2017) Mark K. Schlegel et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65 -mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial

(2017) Stephen Russell et al.   LANCET

Clinical Proof of Concept for a Novel Hepatocyte-Targeting GalNAc-siRNA Conjugate

(2017) Tracy S. Zimmermann et al.   MOLECULAR THERAPY

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

(2017) Norbert Pardi et al.   NATURE

After Glybera's withdrawal, what's next for gene therapy?

(2017) Melanie Senior   NATURE BIOTECHNOLOGY

The chemical evolution of oligonucleotide therapies of clinical utility

(2017) Anastasia Khvorova et al.   NATURE BIOTECHNOLOGY

Cellular uptake and trafficking of antisense oligonucleotides

(2017) Stanley T Crooke et al.   NATURE BIOTECHNOLOGY

Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

(2017) Richard S. Finkel et al.   NEW ENGLAND JOURNAL OF MEDICINE

Impact of enhanced metabolic stability on pharmacokinetics and pharmacodynamics of GalNAc–siRNA conjugates

(2017) Jayaprakash K. Nair et al.   NUCLEIC ACIDS RESEARCH

Antisense oligonucleotides targeting translation inhibitory elements in 5′ UTRs can selectively increase protein levels

(2017) Xue-hai Liang et al.   NUCLEIC ACIDS RESEARCH

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

(2017) Norbert Pardi et al.   Nature Communications

Eteplirsen in the treatment of Duchenne muscular dystrophy

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

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

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

Gene Therapy for “Bubble Boy” Disease

(2016) Jonathan Hoggatt   CELL

Influence of particle size on the in vivo potency of lipid nanoparticle formulations of siRNA

(2016) Sam Chen et al.   JOURNAL OF CONTROLLED RELEASE

Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study

(2016) Richard S Finkel et al.   LANCET

Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial

(2016) Jean Bennett et al.   LANCET

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

(2016) Lena M. Kranz et al.   NATURE

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

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

Results from a phase 1 study of nusinersen (ISIS-SMNRx) in children with spinal muscular atrophy

(2016) Claudia A. Chiriboga et al.   NEUROLOGY

Mutant MHC class II epitopes drive therapeutic immune responses to cancer

(2015) Sebastian Kreiter et al.   NATURE

Gene therapy returns to centre stage

(2015) Luigi Naldini   NATURE

$1-million price tag set for Glybera gene therapy

(2015) Chris Morrison   NATURE BIOTECHNOLOGY

Long-Term Effect of Gene Therapy on Leber’s Congenital Amaurosis

(2015) James W.B. Bainbridge et al.   NEW ENGLAND JOURNAL OF MEDICINE

Improvement and Decline in Vision with Gene Therapy in Childhood Blindness

(2015) Samuel G. Jacobson et al.   NEW ENGLAND JOURNAL OF MEDICINE

2′-Fluoro-modified phosphorothioate oligonucleotide can cause rapid degradation of P54nrb and PSF

(2015) Wen Shen et al.   NUCLEIC ACIDS RESEARCH

Preclinical Development of a Subcutaneous ALAS1 RNAi Therapeutic for Treatment of Hepatic Porphyrias Using Circulating RNA Quantification

(2015) Amy Chan et al.   Molecular Therapy-Nucleic Acids

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

Recombinant Adeno-Associated Virus Utilizes Host Cell Nuclear Import Machinery To Enter the Nucleus

(2014) S. C. Nicolson et al.   JOURNAL OF VIROLOGY

Targeted delivery of antisense oligonucleotides to hepatocytes using triantennaryN-acetyl galactosamine improves potency 10-fold in mice

(2014) Thazha P. Prakash et al.   NUCLEIC ACIDS RESEARCH

AAV8 capsid variable regions at the two-fold symmetry axis contribute to high liver transduction by mediating nuclear entry and capsid uncoating

(2014) Rebeca M. Tenney et al.   VIROLOGY

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

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

Immune Responses to AAV-Vectors, the Glybera Example from Bench to Bedside

(2014) Valerie Ferreira et al.   Frontiers in Immunology

Safety and Efficacy of RNAi Therapy for Transthyretin Amyloidosis

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

Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement

(2013) A. V. Cideciyan et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Influence of Polyethylene Glycol Lipid Desorption Rates on Pharmacokinetics and Pharmacodynamics of siRNA Lipid Nanoparticles

(2013) Barbara L Mui et al.   Molecular Therapy-Nucleic Acids

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

(2012) Muthusamy Jayaraman et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

Amido-Bridged Nucleic Acids (AmNAs): Synthesis, Duplex Stability, Nuclease Resistance, and in Vitro Antisense Potency

(2012) Aiko Yahara et al.   CHEMBIOCHEM

Antisense oligonucleotide therapeutics for inherited neurodegenerative diseases

(2012) Amber L. Southwell et al.   TRENDS IN MOLECULAR MEDICINE

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

(2012) Nathan M Belliveau et al.   Molecular Therapy-Nucleic Acids

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

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

(2010) Akin Akinc et al.   MOLECULAR THERAPY

Efficient and specific knockdown of small non-coding RNAs in mammalian cells and in mice

(2010) Xue-hai Liang et al.   NUCLEIC ACIDS RESEARCH

Worldwide Epidemiology of Neutralizing Antibodies to Adeno‐Associated Viruses

(2009) Roberto Calcedo et al.   JOURNAL OF INFECTIOUS DISEASES

Lessons learned from the gene therapy trial for ornithine transcarbamylase deficiency

(2009) James M. Wilson   MOLECULAR GENETICS AND METABOLISM

Gene Therapy for Immunodeficiency Due to Adenosine Deaminase Deficiency

(2009) Alessandro Aiuti et al.   NEW ENGLAND JOURNAL OF MEDICINE