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Aberrant gene expression and muscular dystrophy: role of non-coding RNAs and alternative splicing in the pathogenesis and potential therapies (NCRNAS-SPLICING-FSHD)
Start date: Sep 1, 2008, End date: Aug 31, 2014,
Our goal is to characterize the molecular pathogenesis of facioscapulohumeral muscular dystrophy (FSHD) in order to develop possible therapeutic approaches. FSHD is the third most important myopathy. Currently, no therapeutic treatment is available for FSHD. Unlike the majority of genetic diseases, FSHD is not caused by mutation in a protein-coding gene. Instead, FSHD is the result of a complex ep ...
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