making muscle wasting history

Exon Skipping

• Last Edited: 6 Apr 2009
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The exon skipping technique tries to change a Duchenne mutation into a Becker mutation. If a variation disturbs the reading frame and thus causes Duchenne dystrophy, the reading frame can be resto...

Tags: exon skipping, exon, antisense oligonucleotide, RNA, duchenne.

Exon skipping for Duchenne Muscular Dystrophy: a successful clinical trial in the UK

• Last Edited: 5 May 2009
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By Professor Francesco Muntoni, UCL Institute of Child Health LondonProfessor Francesco Muntoni of UCL and Head of the MDEX Consortium discusses the results of the latest successful experimental t...

Tags: Exon skipping, AVI BioPharma, AVI4658, antisense oligonucleotide, exon 51.

Update on the Therapeutic approaches to muscular dystrophy

• Last Edited: 10 May 2011
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This is a summary of the article written by Aurelie Goyenvalle, Jane T Seto, Kay E Davies and Jeffrey Chamberlain, which was published by Oxford University Press on 5 April 2011.The article is a r...

Tags: exon skipping, gene therapy, Viral Vector, Dystrophin, rAAV, Antisense Oligonucleotide, PMO, PPMO, Duchenne, muscular dystrophy, Utrophin.