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Gene replacement therapy in a model of Charcot-Marie-Tooth 4C neuropathy
Charcot-Marie-Tooth disease type 4C is the most common recessively inherited demyelinating neuropathy that results from loss of function mutations in the SH3TC2 gene. Sh3tc2(−/−) mice represent a well characterized disease model developing early onset progressive peripheral neuropathy with hypo- and...
Autores principales: | , , , , , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Oxford University Press
2019
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6487329/ https://www.ncbi.nlm.nih.gov/pubmed/30907403 http://dx.doi.org/10.1093/brain/awz064 |