Reversible immortalisation enables genetic correction of human muscle progenitors and engineering of next‐generation human artificial chromosomes for Duchenne muscular dystrophy

Transferring large or multiple genes into primary human stem/progenitor cells is challenged by restrictions in vector capacity, and this hurdle limits the success of gene therapy. A paradigm is Duchenne muscular dystrophy (DMD), an incurable disorder caused by mutations in the largest human gene: dy...

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Detalles Bibliográficos
Autores principales: Benedetti, Sara, Uno, Narumi, Hoshiya, Hidetoshi, Ragazzi, Martina, Ferrari, Giulia, Kazuki, Yasuhiro, Moyle, Louise Anne, Tonlorenzi, Rossana, Lombardo, Angelo, Chaouch, Soraya, Mouly, Vincent, Moore, Marc, Popplewell, Linda, Kazuki, Kanako, Katoh, Motonobu, Naldini, Luigi, Dickson, George, Messina, Graziella, Oshimura, Mitsuo, Cossu, Giulio, Tedesco, Francesco Saverio
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2017
Materias:
Research Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5801502/
https://www.ncbi.nlm.nih.gov/pubmed/29242210
http://dx.doi.org/10.15252/emmm.201607284

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