Safety Pharmacology and Genotoxicity Evaluation of AVI-4658

Efficacy of systemic morpholino exon-skipping in duchenne dystrophy dogs

(2009) Toshifumi Yokota et al.   ANNALS OF NEUROLOGY

In vivocomparison of 2′-O-methyl phosphorothioate and morpholino antisense oligonucleotides for Duchenne muscular dystrophy exon skipping

(2009) Hans A. Heemskerk et al.   JOURNAL OF GENE MEDICINE

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

T.O.4 A phase I/IIa study on antisense compound PRO051 in patients with Duchenne muscular dystrophy

(2009) N.M. Goemans et al.   NEUROMUSCULAR DISORDERS

Therapeutic Potential of Splice-Switching Oligonucleotides

(2009) John Bauman et al.   OLIGONUCLEOTIDES

Antisense, RNAi, and gene silencing strategies for therapy: Mission possible or impossible?

(2008) E RAYBURN et al.   DRUG DISCOVERY TODAY