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...

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Detalhes bibliográficos
Publicado no:Brain
Main Authors: Schiza, Natasa, Georgiou, Elena, Kagiava, Alexia, Médard, Jean-Jacques, Richter, Jan, Tryfonos, Christina, Sargiannidou, Irene, Heslegrave, Amanda J, Rossor, Alexander M, Zetterberg, Henrik, Reilly, Mary M, Christodoulou, Christina, Chrast, Roman, Kleopa, Kleopas A
Formato: Artigo
Idioma:Inglês
Publicado em: Oxford University Press 2019
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Original Articles
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6487329/
https://ncbi.nlm.nih.gov/pubmed/30907403
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/brain/awz064
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