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

• Auteurs principaux: Silvana M G Jirka (Auteur), Christa L Tanganyika-de Winter (Auteur), Joke W Boertje-van der Meulen (Auteur), Maaike van Putten (Auteur), Monika Hiller (Auteur), Rick Vermue (Auteur), Peter C de Visser (Auteur), Annemieke Aartsma-Rus (Auteur)
• Format: Livre
• Publié: Elsevier, 2015-01-01T00:00:00Z.
• Sujets: article
• Accès en ligne: 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...