Evaluation of 2'-Deoxy-2'-fluoro Antisense Oligonucleotides for Exon Skipping in Duchenne Muscular Dystrophy

• Main Authors: Silvana M G Jirka (Author), Christa L Tanganyika-de Winter (Author), Joke W Boertje-van der Meulen (Author), Maaike van Putten (Author), Monika Hiller (Author), Rick Vermue (Author), Peter C de Visser (Author), Annemieke Aartsma-Rus (Author)
• Format: Book
• Published: Elsevier, 2015-01-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Duchenne muscular dystrophy (DMD) is a severe muscle wasting disorder typically caused by frame-shifting mutations in the DMD gene. Restoration of the reading frame would allow the production of a shorter but partly functional dystrophin protein as seen in Becker muscular dystrophy patients. This ca...