Antisense oligonucleotide-based drug development for Cystic Fibrosis patients carrying the 3849+10 kb C-to-T splicing mutation

Optimization of antisense-mediated exon skipping for Duchenne muscular dystrophy

(2020) Kasia Dzierlega et al.   GENE THERAPY

Advances in oligonucleotide drug delivery

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

Antisense oligonucleotide-mediated correction of CFTR splicing improves chloride secretion in cystic fibrosis patient-derived bronchial epithelial cells

(2020) Wren E Michaels et al.   NUCLEIC ACIDS RESEARCH

Take it personally: how personal we reach when we are so different from each other?

(2019) Eitan Kerem et al.   Journal of Cystic Fibrosis

The future of cystic fibrosis care: a global perspective

(2019) Scott C Bell et al.   Lancet Respiratory Medicine

Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial

(2019) Harry G M Heijerman et al.   LANCET

Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele

(2019) Peter G. Middleton et al.   NEW ENGLAND JOURNAL OF MEDICINE

CFTR modulator theratyping: Current status, gaps and future directions

(2018) John Paul Clancy et al.   Journal of Cystic Fibrosis

Advances in therapy for spinal muscular atrophy: promises and challenges

(2018) Ewout J. N. Groen et al.   Nature Reviews Neurology

Inhaled ENaC antisense oligonucleotide ameliorates cystic fibrosis-like lung disease in mice

(2017) Jeff R. Crosby et al.   Journal of Cystic Fibrosis

Oligonucleotide Therapies for the Lung: Ready to Return to the Clinic?

(2017) Manish Kumar et al.   MOLECULAR THERAPY

The chemical evolution of oligonucleotide therapies of clinical utility

(2017) Anastasia Khvorova et al.   NATURE BIOTECHNOLOGY

Tezacaftor–Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis

(2017) Steven M. Rowe et al.   NEW ENGLAND JOURNAL OF MEDICINE

Correction of CFTR function in nasal epithelial cells from cystic fibrosis patients predicts improvement of respiratory function by CFTR modulators

(2017) Iwona M. Pranke et al.   Scientific Reports

Big win possible for Ionis/Biogen antisense drug in muscular atrophy

(2016) Ken Garber   NATURE BIOTECHNOLOGY

Pharmacokinetics, biodistribution and cell uptake of antisense oligonucleotides

(2015) Richard S. Geary et al.   ADVANCED DRUG DELIVERY REVIEWS

Correction of a Cystic Fibrosis Splicing Mutation by Antisense Oligonucleotides

(2015) Susana Igreja et al.   HUMAN MUTATION

Local and systemic tolerability of a 2′O-methoxyethyl antisense oligonucleotide targeting interleukin-4 receptor-α delivery by inhalation in mouse and monkey

(2014) Robert A. Fey et al.   INHALATION TOXICOLOGY

Comparison of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) and Ciliary Beat Frequency Activation by the CFTR Modulators Genistein, VRT-532, and UCCF-152 in Primary Sinonasal Epithelial Cultures

(2014) Bryant T. Conger et al.   JAMA Otolaryngology-Head & Neck Surgery

Synthetic Aminoglycosides Efficiently Suppress Cystic Fibrosis Transmembrane Conductance Regulator Nonsense Mutations and Are Enhanced by Ivacaftor

(2013) Xiaojiao Xue et al.   AMERICAN JOURNAL OF RESPIRATORY CELL AND MOLECULAR BIOLOGY

Assessing the Disease-Liability of Mutations in CFTR

(2013) C. Ferec et al.   Cold Spring Harbor Perspectives in Medicine

RNA therapeutics: beyond RNA interference and antisense oligonucleotides

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

A Pharmacologic Approach to Acquired Cystic Fibrosis Transmembrane Conductance Regulator Dysfunction in Smoking Related Lung Disease

(2012) Peter A. Sloane et al.   PLoS One

Selection, Optimization, and Pharmacokinetic Properties of a Novel, Potent Antiviral Locked Nucleic Acid-Based Antisense Oligomer Targeting Hepatitis C Virus Internal Ribosome Entry Site

(2011) Carl Laxton et al.   ANTIMICROBIAL AGENTS AND CHEMOTHERAPY

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

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

Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model

(2010) Y. Hua et al.   GENES & DEVELOPMENT

Advantageous toxicity profile of inhaled antisense oligonucleotides following chronic dosing in non-human primates

(2008) Alain Guimond et al.   PULMONARY PHARMACOLOGY & THERAPEUTICS