Autologous skeletal muscle derived cells expressing a novel functional dystrophin provide a potential therapy for Duchenne Muscular Dystrophy

Autologous stem cells that have been genetically modified to express dystrophin are a possible means of treating Duchenne Muscular Dystrophy (DMD). To maximize the therapeutic effect, dystrophin construct needs to contain as many functional motifs as possible, within the packaging capacity of the vi...

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Detalhes bibliográficos
Publicado no:Sci Rep
Main Authors: Meng, Jinhong, Counsell, John R., Reza, Mojgan, Laval, Steven H., Danos, Olivier, Thrasher, Adrian, Lochmüller, Hanns, Muntoni, Francesco, Morgan, Jennifer E.
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2016
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4728433/
https://ncbi.nlm.nih.gov/pubmed/26813695
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/srep19750
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