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

• Autori principali: Silvana M G Jirka (Autore), Christa L Tanganyika-de Winter (Autore), Joke W Boertje-van der Meulen (Autore), Maaike van Putten (Autore), Monika Hiller (Autore), Rick Vermue (Autore), Peter C de Visser (Autore), Annemieke Aartsma-Rus (Autore)
• Natura: Libro
• Pubblicazione: Elsevier, 2015-01-01T00:00:00Z.
• Soggetti: article
• Accesso online: 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...