Restoration of the Dystrophin-associated Glycoprotein Complex After Exon Skipping Therapy in Duchenne Muscular Dystrophy

We previously conducted a proof of principle; dose escalation study in Duchenne muscular dystrophy (DMD) patients using the morpholino splice-switching oligonucleotide AVI-4658 (eteplirsen) that induces skipping of dystrophin exon 51 in patients with relevant deletions, restores the open reading fra...

Full description

Saved in:
Bibliographic Details
Main Authors: Cirak, Sebahattin, Feng, Lucy, Anthony, Karen, Arechavala-Gomeza, Virginia, Torelli, Silvia, Sewry, Caroline, Morgan, Jennifer E, Muntoni, Francesco
Format: Artigo
Language:Inglês
Published: Nature Publishing Group 2012
Subjects:
Original Article
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC3277241/
https://ncbi.nlm.nih.gov/pubmed/22086232
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2011.248
Tags: Add Tag
No Tags, Be the first to tag this record!

Similar Items