Targeted Exon Skipping to Address "Leaky" Mutations in the Dystrophin Gene

• Main Authors: Sue Fletcher (Author), Carl F. Adkin (Author), Penny Meloni (Author), Brenda Wong (Author), Francesco Muntoni (Author), Ryszard Kole (Author), Clayton Fragall (Author), Kane Greer (Author), Russell Johnsen (Author), Steve D. Wilton (Author)
• Format: Book
• Published: Elsevier, 2012-01-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...