Toxicological Characterization of Exon Skipping Phosphorodiamidate Morpholino Oligomers (PMOs) in Non-human Primates
Author:
Affiliation:
1. R&D Pharmacology/Toxicology, Sarepta Therapeutics, Inc., Cambridge, MA, USA
2. Covance Laboratories, Inc., Madison, WI, USA
3. Research and Innovation, Sarepta Therapeutics, Inc., Cambridge, MA, USA
Publisher
IOS Press
Subject
Clinical Neurology,Neurology
Reference34 articles.
1. Splicing therapy for neuromuscular disease;Douglas;Mol Cell Neurosci,2013
2. Report of MDA muscle disease symposium on newborn screening for Duchenne muscular dystrophy;Mendell;Muscle Nerve,2013
3. Targeting mRNA splicing as a potential treatment for Duchenne muscular dystrophy;Kole;Discov Med,2012
4. Targeting RNA to treat neuromuscular disease;Muntoni;Nat Rev Drug Discov,2011
5. Dystrophin and mutations: One gene, several proteins, multiple phenotypes;Muntoni;Lancet Neurol,2003
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