Antisense Oligonucleotides: An Emerging Area in Drug Discovery and Development

Givosiran: First Approval

(2020) Lesley J. Scott   DRUGS

Increased dystrophin production with golodirsen in patients with Duchenne muscular dystrophy

(2020) Diane E. Frank et al.   NEUROLOGY

STUDY DESIGN AND RATIONALE OF HELIOS-B: A PHASE 3 STUDY TO EVALUATE THE CLINICAL EFFICACY AND SAFETY OF VUTRISIRAN IN PATIENTS WITH ATTR AMYLOIDOSIS WITH CARDIOMYOPATHY

(2020) Rebecca Shilling et al.   JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY

Inclisiran for the Treatment of Heterozygous Familial Hypercholesterolemia

(2020) Frederick J. Raal et al.   NEW ENGLAND JOURNAL OF MEDICINE

Oligonucleotides and the COVID-19 Pandemic: A Perspective

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

Research and Development on Therapeutic Agents and Vaccines for COVID-19 and Related Human Coronavirus Diseases

(2020) Cynthia Liu et al.   ACS Central Science

Targeting RNA : A Transformative Therapeutic Strategy

(2019) Wei Yin et al.   CTS-Clinical and Translational Science

Small interfering RNAs (siRNAs) in cancer therapy: a nano-based approach

(2019) Ghanbar Mahmoodi Chalbatani et al.   International Journal of Nanomedicine

Cellular functions of long noncoding RNAs

(2019) Run-Wen Yao et al.   NATURE CELL BIOLOGY

Targeting Huntingtin Expression in Patients with Huntington’s Disease

(2019) Sarah J. Tabrizi et al.   NEW ENGLAND JOURNAL OF MEDICINE

Hereditary transthyretin amyloidosis: a model of medical progress for a fatal disease

(2019) David Adams et al.   Nature Reviews Neurology

The biogenesis, biology and characterization of circular RNAs

(2019) Lasse S. Kristensen et al.   NATURE REVIEWS GENETICS

Volanesorsen and Triglyceride Levels in Familial Chylomicronemia Syndrome

(2019) Joseph L. Witztum et al.   NEW ENGLAND JOURNAL OF MEDICINE

The Role of Non-coding RNAs in Oncology

(2019) Frank J. Slack et al.   CELL

Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease

(2019) Jinkuk Kim et al.   NEW ENGLAND JOURNAL OF MEDICINE

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

(2019) Akin Akinc et al.   Nature Nanotechnology

RNA-Targeted Therapeutics

(2018) Stanley T. Crooke et al.   Cell Metabolism

Locked nucleic acid: modality, diversity, and drug discovery

(2018) Peter H. Hagedorn et al.   DRUG DISCOVERY TODAY

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

Acute hepatotoxicity of 2′ fluoro-modified 5–10–5 gapmer phosphorothioate oligonucleotides in mice correlates with intracellular protein binding and the loss of DBHS proteins

(2018) Wen Shen et al.   NUCLEIC ACIDS RESEARCH

Lipid Nanoparticles Enabling Gene Therapies: From Concepts to Clinical Utility

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

Cobomarsen, an oligonucleotide inhibitor of miR-155, co-ordinately regulates multiple survival pathways to reduce cellular proliferation and survival in cutaneous T-cell lymphoma

(2018) Anita G. Seto et al.   BRITISH JOURNAL OF HAEMATOLOGY

Intrathecal administration of nusinersen in adolescent and adult SMA type 2 and 3 patients

(2018) Claudia D. Wurster et al.   JOURNAL OF NEUROLOGY

Intravascular hemolysis and the pathophysiology of sickle cell disease

(2017) Gregory J. Kato et al.   JOURNAL OF CLINICAL INVESTIGATION

The chemical evolution of oligonucleotide therapies of clinical utility

(2017) Anastasia Khvorova et al.   NATURE BIOTECHNOLOGY

Overcoming cellular barriers for RNA therapeutics

(2017) Steven F Dowdy   NATURE BIOTECHNOLOGY

Antisense oligonucleotides: the next frontier for treatment of neurological disorders

(2017) Carlo Rinaldi et al.   Nature Reviews Neurology

Porphyria

(2017) D. Montgomery Bissell et al.   NEW ENGLAND JOURNAL OF MEDICINE

Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

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

Eteplirsen in the treatment of Duchenne muscular dystrophy

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

FDA Approves Eteplirsen for Duchenne Muscular Dystrophy: The Next Chapter in the Eteplirsen Saga

(2017) Annemieke Aartsma-Rus et al.   Nucleic Acid Therapeutics

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

(2017) James C. Kaczmarek et al.   Genome Medicine

Antisense Oligonucleotides Promote Exon Inclusion and Correct the Common c.-32-13T>G GAA Splicing Variant in Pompe Disease

(2017) Erik van der Wal et al.   Molecular Therapy-Nucleic Acids

Gapmer Antisense Oligonucleotides Suppress the Mutant Allele of COL6A3 and Restore Functional Protein in Ullrich Muscular Dystrophy

(2017) Elena Marrosu et al.   Molecular Therapy-Nucleic Acids

Preclinical and Clinical Advances of GalNAc-Decorated Nucleic Acid Therapeutics

(2017) Yuanyu Huang   Molecular Therapy-Nucleic Acids

PLGA-based dual targeted nanoparticles enhance miRNA transfection efficiency in hepatic carcinoma

(2017) Chenlei Cai et al.   Scientific Reports

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

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

MicroRNA in Control of Gene Expression: An Overview of Nuclear Functions

(2016) Caterina Catalanotto et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Insights on the pathophysiology of Alzheimer's disease: The crosstalk between amyloid pathology, neuroinflammation and the peripheral immune system

(2016) Sandro Dá Mesquita et al.   NEUROSCIENCE AND BIOBEHAVIORAL REVIEWS

The delivery of therapeutic oligonucleotides

(2016) Rudolph L. Juliano   NUCLEIC ACIDS RESEARCH

microRNA Therapeutics in Cancer — An Emerging Concept

(2016) Maitri Y. Shah et al.   EBioMedicine

Motor neuron cell-nonautonomous rescue of spinal muscular atrophy phenotypes in mild and severe transgenic mouse models

(2015) Yimin Hua et al.   GENES & DEVELOPMENT

Going beyond the liver: Progress and challenges of targeted delivery of siRNA therapeutics

(2015) Cornelia Lorenzer et al.   JOURNAL OF CONTROLLED RELEASE

Transthyretin (ATTR) amyloidosis: clinical spectrum, molecular pathogenesis and disease-modifying treatments

(2015) Yoshiki Sekijima   JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY

Increased brain uptake of targeted nanoparticles by adding an acid-cleavable linkage between transferrin and the nanoparticle core

(2015) Andrew J. Clark et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Long-circulating siRNA nanoparticles for validating Prohibitin1-targeted non-small cell lung cancer treatment

(2015) Xi Zhu et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

In VitroAntiviral Activity and Preclinical and Clinical Resistance Profile of Miravirsen, a Novel Anti-Hepatitis C Virus Therapeutic Targeting the Human Factor miR-122

(2014) Søren Ottosen et al.   ANTIMICROBIAL AGENTS AND CHEMOTHERAPY

Anti–microRNA-21 oligonucleotides prevent Alport nephropathy progression by stimulating metabolic pathways

(2014) Ivan G. Gomez et al.   JOURNAL OF CLINICAL INVESTIGATION

Nonsense-mediated decay as a terminating mechanism for antisense oligonucleotides

(2014) Amanda J. Ward et al.   NUCLEIC ACIDS RESEARCH

Antisense oligonucleotides: modifications and clinical trials

(2014) Vivek K. Sharma et al.   MedChemComm

Exon-Skipping Antisense Oligonucleotides to Correct Missplicing in Neurogenetic Diseases

(2014) Kavitha Siva et al.   Nucleic Acid Therapeutics

Phosphorothioates, Essential Components of Therapeutic Oligonucleotides

(2014) Fritz Eckstein   Nucleic Acid Therapeutics

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

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

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

Mipomersen Sodium: First Global Approval

(2013) Philip Hair et al.   DRUGS

Expression of Asialoglycoprotein Receptor 1 in Human Hepatocellular Carcinoma

(2013) Bin Shi et al.   JOURNAL OF HISTOCHEMISTRY & CYTOCHEMISTRY

Targeting RNA splicing for disease therapy

(2013) Mallory A. Havens et al.   Wiley Interdisciplinary Reviews-RNA

PLGA-based nanoparticles: An overview of biomedical applications

(2012) Fabienne Danhier et al.   JOURNAL OF CONTROLLED RELEASE

The Possible “Proton Sponge ” Effect of Polyethylenimine (PEI) Does Not Include Change in Lysosomal pH

(2012) Rikke V Benjaminsen et al.   MOLECULAR THERAPY

Spinal Muscular Atrophy

(2011) Stephen J. Kolb   ARCHIVES OF NEUROLOGY

Strand Invasion of Mixed-Sequence, Double-Helical B-DNA by γ-Peptide Nucleic Acids Containing G-Clamp Nucleobases under Physiological Conditions

(2011) Srinivas Rapireddy et al.   BIOCHEMISTRY

Familial Hypercholesterolemia: Screening, diagnosis and management of pediatric and adult patients

(2011) Anne C. Goldberg et al.   Journal of Clinical Lipidology

Silencing disease genes in the laboratory and the clinic

(2011) Jonathan K Watts et al.   JOURNAL OF PATHOLOGY

Pseudogenes: Pseudo-functional or key regulators in health and disease?

(2011) R. C. Pink et al.   RNA

Antisense Oligonucleotides Delivered to the Mouse CNS Ameliorate Symptoms of Severe Spinal Muscular Atrophy

(2011) M. A. Passini et al.   Science Translational Medicine

Huntington's Disease

(2011) S. Finkbeiner   Cold Spring Harbor Perspectives in Biology

RNA Targeting Therapeutics: Molecular Mechanisms of Antisense Oligonucleotides as a Therapeutic Platform

(2010) C. Frank Bennett et al.   Annual Review of Pharmacology and Toxicology

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

Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study

(2009) Maria Kinali et al.   LANCET NEUROLOGY

Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management

(2009) Katharine Bushby et al.   LANCET NEUROLOGY

Therapeutic Potential of Splice-Switching Oligonucleotides

(2009) John Bauman et al.   OLIGONUCLEOTIDES

RNA repair restores hemoglobin expression in IVS2-654 thalassemic mice

(2009) S. Svasti et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Oligonucleotide antiviral therapeutics: Antisense and RNA interference for highly pathogenic RNA viruses

(2008) Kevin B. Spurgers et al.   ANTIVIRAL RESEARCH

Distinct Uptake Routes of Cell-Penetrating Peptide Conjugates

(2008) Per Lundin et al.   BIOCONJUGATE CHEMISTRY

A Simple Cytosine to G-Clamp Nucleobase Substitution Enables Chiral γ-PNAs to Invade Mixed-Sequence Double-Helical B-form DNA

(2008) Venugopal Chenna et al.   CHEMBIOCHEM

Toward a more complete view of tRNA biology

(2008) Richard Giegé   NATURE STRUCTURAL & MOLECULAR BIOLOGY