http://www.ncbi.nlm.nih.gov/pubmed/19006096

J Gene Med. 2008 Nov 12. [Epub ahead of print]

By-passing the nonsense mutation in the 4(CV) mouse model of muscular dystrophy by induced exon skipping.

Mitrpant C, Fletcher S, Iversen PL, Wilton SD.

Abstract excerpts:

“Both exons 52 and 53 must be excised to remove the mutation and maintain the reading frame.”

“PMO cocktails could restore synthesis of near-full length dystrophin protein in cultured 4(CV) myogenic cells and in vivo, after a single intramuscular injection.”

“As in previous studies, the stability and efficiency of PMOs proved superior to 2OMeAOs for consistent and sustained protein induction in vivo.”