Title : Long-term rescue of dystrophin expression and improvement in muscle pathology and function in dystrophic mdx mice by peptide-conjugated morpholino.

Pub. Date : 2012 Aug

PMID : 22683468






1 Functional Relationships(s)
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1 Phase 2 clinical trials in the United Kingdom and the Netherlands have reported induction of dystrophin expression in muscle of Duchenne muscular dystrophy patients by systemic administration of both phosphorodiamidate morpholino oligomers (PMO) and 2"-O-methyl phosphorothioate. 2"-o-methyl phosphorothioate dystrophin Homo sapiens