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Selection-free gene repair after adenoviral vector transduction of designer nucleases: rescue of dystrophin synthesis in DMD muscle cell populations

Duchenne muscular dystrophy (DMD) is a fatal X-linked muscle-wasting disorder caused by mutations in the 2.4 Mb dystrophin-encoding DMD gene. The integration of gene delivery and gene editing technologies based on viral vectors and sequence-specific designer nucleases, respectively, constitutes a po...

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Detalles Bibliográficos
Autores principales:	Maggio, Ignazio, Stefanucci, Luca, Janssen, Josephine M., Liu, Jin, Chen, Xiaoyu, Mouly, Vincent, Gonçalves, Manuel A.F.V.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Oxford University Press 2016
Materias:	Synthetic Biology and Bioengineering
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4756843/ https://www.ncbi.nlm.nih.gov/pubmed/26762977 http://dx.doi.org/10.1093/nar/gkv1540

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