Targeted Exon Skipping to Address “Leaky” Mutations in the Dystrophin Gene

Targeted Exon Skipping to Address “Leaky” Mutations in the Dystrophin Gene

Protein-truncating mutations in the dystrophin gene lead to the progressive muscle wasting disorder Duchenne muscular dystrophy, whereas in-frame deletions typically manifest as the milder allelic condition, Becker muscular dystrophy. Antisense oligomer-induced exon skipping can modify dystrophin ge...

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Bibliographic Details
Main Authors: Sue Fletcher, Carl F. Adkin, Penny Meloni, Brenda Wong, Francesco Muntoni, Ryszard Kole, Clayton Fragall, Kane Greer, Russell Johnsen, Steve D. Wilton
Format: Article
Language:English
Published: Elsevier 2012-01-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
antisense oligomers
Duchenne muscular dystrophy
exon skipping
personalized genetic therapy
splice-switching
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253116301068

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http://www.sciencedirect.com/science/article/pii/S2162253116301068

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