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Article

RNA-targeted splice-correction therapy for neuromuscular disease

11 Feb 2010

Abstract excerpt

Splice-modulation therapy, whereby molecular manipulation of premessenger RNA splicing is engineered to yield genetic correction, is a promising novel therapy for genetic diseases of muscle and nerve-the prototypical example being Duchenne muscular dystrophy. Duchenne muscular dystrophy is the most common childhood genetic disease, affecting one in 3500 newborn boys, causing progressive muscle weakness, heart and...

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PMID: 20150322
DOI: 10.1093/brain/awq002

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