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

• Asıl Yazarlar: Silvana M G Jirka (Yazar), Christa L Tanganyika-de Winter (Yazar), Joke W Boertje-van der Meulen (Yazar), Maaike van Putten (Yazar), Monika Hiller (Yazar), Rick Vermue (Yazar), Peter C de Visser (Yazar), Annemieke Aartsma-Rus (Yazar)
• Materyal Türü: Kitap
• Baskı/Yayın Bilgisi: Elsevier, 2015-01-01T00:00:00Z.
• Konular: article
• Online Erişim: 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...