Systemic Antisense Therapeutics for Dystrophin and Myostatin Exon Splice Modulation Improve Muscle Pathology of Adult mdx Mice

• Main Authors: Ngoc Lu-Nguyen (Author), Alberto Malerba (Author), Linda Popplewell (Author), Fred Schnell (Author), Gunnar Hanson (Author), George Dickson (Author)
• Format: Book
• Published: Elsevier, 2017-03-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Antisense-mediated exon skipping is a promising approach for the treatment of Duchenne muscular dystrophy (DMD), a rare life-threatening genetic disease due to dystrophin deficiency. Such an approach can restore the disrupted reading frame of dystrophin pre-mRNA, generating a truncated form of the p...