Article
Comparative analysis of antisense oligonucleotide sequences targeting exon 53 of the human DMD gene: Implications for future clinical trials.
Neuromuscular disorders : NMD - 1 Feb 2010
Popplewell Linda J, Adkin Carl, Arechavala-Gomeza Virginia, Aartsma-Rus Annemieke, de Winter Christa L, Wilton Steve D, Morgan Jennifer E, Muntoni Francesco, Graham Ian R, Dickson George
Abstract excerpt
Duchenne muscular dystrophy (DMD) is caused by the lack of functional dystrophin protein, most commonly as a result of a range of out-of-frame mutations in the DMD gene. Modulation of pre-mRNA splicing with antisense oligonucleotides (AOs) to restore the reading frame has been demonstrated in vit...
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