«We took patient - derived cells that had the most common mutation responsible for Duchenne muscular dystrophy and we corrected them in vitro to restore production of the missing dystrophin protein in the cells. (sciencedaily.com)
Correction of dystrophin expression in cells from Duchenne muscular dystrophy patients through genomic excision of exon 51 by zinc finger nucleases. (blog.addgene.org)
«The very slight increases in dystrophin production didn't seem to me reasonably likely to predict the possibility of clinical benefit,» says internist Aaron Kesselheim of Boston's Brigham and Women's Hospital, who was one of the FDA advisory committee members who voted against recommending approval of eteplirsen. (protomag.com)