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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Bibliografski detalji
Izdano u:Sci Rep
Glavni autori: Meng, Jinhong, Counsell, John R., Reza, Mojgan, Laval, Steven H., Danos, Olivier, Thrasher, Adrian, Lochmüller, Hanns, Muntoni, Francesco, Morgan, Jennifer E.
Format: Artigo
Jezik:Inglês
Izdano: Nature Publishing Group 2016
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Article
Online pristup: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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