AAV1.NT-3 Gene Therapy for Charcot–Marie–Tooth Neuropathy

Charcot–Marie–Tooth (CMT) neuropathies represent a heterogeneous group of peripheral nerve disorders affecting 1 in 2,500 persons. One variant, CMT1A, is a primary Schwann cell (SC) disorder, and represents the single most common variant. In previous studies, we showed that neurotrophin-3 (NT-3) imp...

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Autors principals: Sahenk, Zarife, Galloway, Gloria, Clark, Kelly Reed, Malik, Vinod, Rodino-Klapac, Louise R, Kaspar, Brian K., Chen, Lei, Braganza, Cilwyn, Montgomery, Chrystal, Mendell, Jerry R
Format: Artigo
Idioma:Inglês
Publicat: Nature Publishing Group 2014
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Original Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC3944324/
https://ncbi.nlm.nih.gov/pubmed/24162799
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2013.250
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