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Splice-Switching Therapy for Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) is a genetic disorder with severity ranging from premature death in infants to restricted motor function in adult life. Despite the genetic cause of this disease being known for over twenty years, only recently has a therapy been approved to treat the most severe form o...

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Detalles Bibliográficos
Autores principales:	Meijboom, Katharina E., Wood, Matthew J.A., McClorey, Graham
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	MDPI 2017
Materias:	Review
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5485525/ https://www.ncbi.nlm.nih.gov/pubmed/28604635 http://dx.doi.org/10.3390/genes8060161

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