Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study. / Kinali, Maria; Arechavala-Gomeza, Virginia; Feng, Lucy; Cirak, Sebahattin; Hunt, David; Adkin, Carl; Guglieri, Michela; Ashton, Emma; Abbs, Stephen; Nihoyannopoulos, Petros; Garralda, Maria Elena; Rutherford, Mary; McCulley, Caroline; Popplewell, Linda; Graham, Ian R; Dickson, George; Wood, Matthew J A; Wells, Dominic J; Wilton, Steve D; Kole, Ryszard; Straub, Volker; Bushby, Kate; Sewry, Caroline; Morgan, Jennifer E; Muntoni, Francesco.
In: Lancet Neurology, Vol. 8, No. 10, 2009, p. 918-28.Research output: Contribution to journal › Article
Published
Abstract
Mutations that disrupt the open reading frame and prevent full translation of DMD, the gene that encodes dystrophin, underlie the fatal X-linked disease Duchenne muscular dystrophy. Oligonucleotides targeted to splicing elements (splice switching oligonucleotides) in DMD pre-mRNA can lead to exon skipping, restoration of the open reading frame, and the production of functional dystrophin in vitro and in vivo, which could benefit patients with this disorder.
| Original language | English |
|---|---|
| Number of pages | 11 |
| Pages | 918-28 |
| Journal | Lancet Neurology |
| Journal publication date | 2009 |
| Journal number | 10 |
| Volume | 8 |
| DOIs | |
| State | Published |
ID: 961812