The Status of Exon Skipping as a Therapeutic Approach to Duchenne Muscular Dystrophy.
Qi-Long Lu, Toshifumi Yokota, Shin'ichi Takeda, Luis Garcia, Francesco Muntoni, Terence Partridge.
Mol Ther. 2011 January 4; 19(1): 9–15. Published online 2010 October 26. doi: 10.1038/mt.2010.219 [Only registered users see links. ]
The Morpholino antisense oligo from Sarepta Therapeutics targeting skipping of exon 51 in dystrophin and intended for treatment of some mutations causing Duchenne muscular dystrophy (DMD) is now listed on clinicaltrials.gov for a phase 3 trial. [Only registered users see links. ]