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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Publicado en: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: Nature Publishing Group 2016
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Article
Acceso en liña: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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