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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...
Autores principales: | 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 |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Nature Publishing Group
2014
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3944324/ https://www.ncbi.nlm.nih.gov/pubmed/24162799 http://dx.doi.org/10.1038/mt.2013.250 |
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