What Can We Learn From Clinical Trials of Exon Skipping for DMD?

Author:

Lu Qi-long,Cirak Sebahattin,Partridge Terence

Publisher

Elsevier BV

Subject

Drug Discovery,Molecular Medicine

Reference22 articles.

1. http://www.gsk.com/media/press-releases/2013/gsk-and-prosensa-announce-primary-endpoint-not-met-in-phase-iii-.html

2. Sarepta Therapeutics http://investorrelations.sareptatherapeutics.com/phoenix.zhtml?c=64231&p=irol-newsArticle&ID=1875187&highlight=

3. In vivo comparison of 2’-O-methyl phosphorothioate and morpholino antisense oligonucleotides for Duchenne muscular dystrophy exon skipping;Heemskerk;J Gene Med,2009

4. Long-term exon skipping studies with 2’-O-methyl phosphorothioate antisense oligonucleotides in dystrophic mouse models;Tanganyika-de Winter;Mol Ther Nucleic Acids,2012

5. Systemic delivery of antisense oligoribonucleotide restores dystrophin expression in body-wide skeletal muscles;Lu;Proc Natl Acad Sci USA.,2005

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