Estimated number of off-target candidate sites for antisense oligonucleotides in human mRNA sequences

FDA-Approved Oligonucleotide Therapies in 2017

(2017) Cy A. Stein et al.   MOLECULAR THERAPY

Nusinersen, an antisense oligonucleotide drug for spinal muscular atrophy

(2017) David R Corey   NATURE NEUROSCIENCE

RefSeq: an update on prokaryotic genome annotation and curation

(2017) Daniel H Haft et al.   NUCLEIC ACIDS RESEARCH

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

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

Strategies for In Vivo Screening and Mitigation of Hepatotoxicity Associated with Antisense Drugs

(2017) Piotr J. Kamola et al.   Molecular Therapy-Nucleic Acids

LNA/DNA mixmer-based antisense oligonucleotides correct alternative splicing of the SMN2 gene and restore SMN protein expression in type 1 SMA fibroblasts

(2017) Aleksander Touznik et al.   Scientific Reports

Splice-switching antisense oligonucleotides as therapeutic drugs

(2016) Mallory A. Havens et al.   NUCLEIC ACIDS RESEARCH

Ribonuclease H1-dependent hepatotoxicity caused by locked nucleic acid-modified gapmer antisense oligonucleotides

(2016) Takeshi Kasuya et al.   Scientific Reports

An overview of the clinical application of antisense oligonucleotides for RNA-targeting therapies

(2015) Graham McClorey et al.   CURRENT OPINION IN PHARMACOLOGY

In silicoandin vitroevaluation of exonic and intronic off-target effects form a critical element of therapeutic ASO gapmer optimization

(2015) Piotr J. Kamola et al.   NUCLEIC ACIDS RESEARCH

Hepatotoxicity of high affinity gapmer antisense oligonucleotides is mediated by RNase H1 dependent promiscuous reduction of very long pre-mRNA transcripts

(2015) Sebastien A. Burel et al.   NUCLEIC ACIDS RESEARCH

CRISPRdirect: software for designing CRISPR/Cas guide RNA with reduced off-target sites

(2014) Yuki Naito et al.   BIOINFORMATICS

Design and evaluation of locked nucleic acid-based splice-switching oligonucleotidesin vitro

(2014) Takenori Shimo et al.   NUCLEIC ACIDS RESEARCH

Antisense Oligonucleotide Therapies

(2014) Kendall S. Frazier   TOXICOLOGIC PATHOLOGY

Mipomersen, an Apolipoprotein B Synthesis Inhibitor, Reduces Atherogenic Lipoproteins in Patients With Severe Hypercholesterolemia at High Cardiovascular Risk

(2013) Gregory S. Thomas et al.   JOURNAL OF THE AMERICAN COLLEGE OF CARDIOLOGY

Assessing unintended hybridization-induced biological effects of oligonucleotides

(2012) Morten Lindow et al.   NATURE BIOTECHNOLOGY

RNA therapeutics: beyond RNA interference and antisense oligonucleotides

(2012) Ryszard Kole et al.   NATURE REVIEWS DRUG DISCOVERY

Antisense drug discovery and development

(2011) Tsuyoshi Yamamoto et al.   Future Medicinal Chemistry