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Development of a strategy to treat limb-girdle muscular dystrophy (LGMD2A) using combined cell and gene therapy strategies (LGMD2A)
Start date: Jul 1, 2010, End date: Nov 29, 2014,
In Duchenne muscular dystrophy, several mutations can be corrected by the exon-skipping technique on cells. In limb-girdle muscular dystrophy type 2A (LGMD2A), however, all the mutations described to date do not seem to be good candidates for the RNA correction technology. The recently identified c.1782+1072 G>C mutation in LGMD2A patients could be potentially repaired at RNA level. The princip ...
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