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Full-length Dysferlin Transfer by the Hyperactive Sleeping Beauty Transposase Restores Dysferlin-deficient Muscle

Dysferlin-deficient muscular dystrophy is a progressive disease characterized by muscle weakness and wasting for which there is no treatment. It is caused by mutations in DYSF, a large, multiexonic gene that forms a coding sequence of 6.2 kb. Sleeping Beauty (SB) transposon is a nonviral gene transf...

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Detalles Bibliográficos
Autores principales:	Escobar, Helena, Schöwel, Verena, Spuler, Simone, Marg, Andreas, Izsvák, Zsuzsanna
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Nature Publishing Group 2016
Materias:	Original Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5012550/ https://www.ncbi.nlm.nih.gov/pubmed/26784637 http://dx.doi.org/10.1038/mtna.2015.52

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