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Rescue of severely affected dystrophin/utrophin-deficient mice through scAAV-U7snRNA-mediated exon skipping

Duchenne muscular dystrophy (DMD) is a severe neuromuscular disorder caused by mutations in the dystrophin gene that result in the absence of functional protein. Antisense-mediated exon skipping is one of the most promising approaches for the treatment of DMD and recent clinical trials have demonstr...

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Detalles Bibliográficos
Autores principales:	Goyenvalle, Aurélie, Babbs, Arran, Wright, Jordan, Wilkins, Vivienne, Powell, Dave, Garcia, Luis, Davies, Kay E.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Oxford University Press 2012
Materias:	Articles
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3349427/ https://www.ncbi.nlm.nih.gov/pubmed/22388933 http://dx.doi.org/10.1093/hmg/dds082

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