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Article

Splicing modulation therapy in the treatment of genetic diseases

1 Dec 2014

Abstract excerpt

Antisense-mediated splicing modulation is a tool that can be exploited in several ways to provide a potential therapy for rare genetic diseases. This approach is currently being tested in clinical trials for Duchenne muscular dystrophy and spinal muscular atrophy. The present review outlines the versatility of the approach to correct cryptic splicing, modulate alternative splicing, restore the open reading frame,...

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PMID: 25506237
DOI: 10.2147/tacg.s71506

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