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Combination of stem cell and gene therapy ameliorates symptoms in Huntington’s disease mice

Huntington’s disease (HD) is a dominantly inherited monogenetic disorder characterized by motor and cognitive dysfunction due to neurodegeneration. The disease is caused by the polyglutamine (polyQ) expansion at the 5′ terminal of the exon 1 of the huntingtin (HTT) gene, IT15, which results in the a...

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Detalhes bibliográficos
Publicado no:NPJ Regen Med
Main Authors: Cho, In Ki, Hunter, Carissa Emerson, Ye, Sarah, Pongos, Alvince Learnz, Chan, Anthony Wing Sang
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group UK 2019
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6435637/
https://ncbi.nlm.nih.gov/pubmed/30937182
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41536-019-0066-7
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