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Splicing modulation therapy in the treatment of genetic diseases

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 ver...

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Detalles Bibliográficos
Autores principales:	Arechavala-Gomeza, Virginia, Khoo, Bernard, Aartsma-Rus, Annemieke
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Dove Medical Press 2014
Materias:	Review
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4259397/ https://www.ncbi.nlm.nih.gov/pubmed/25506237 http://dx.doi.org/10.2147/TACG.S71506

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