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SUPT4H1-edited stem cell therapy rescues neuronal dysfunction in a mouse model for Huntington’s disease

Huntington’s disease (HD) is a severe inherited neurological disorder caused by a CAG repeat expansion in the huntingtin gene (HTT), leading to the accumulation of mutant huntingtin with polyglutamine repeats. Despite its severity, there is no cure for this debilitating disease. HTT lowering strateg...

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Detalles Bibliográficos
Autores principales: Park, Hyun Jung, Han, Areum, Kim, Ji Yeon, Choi, Jiwoo, Bae, Hee Sook, Cho, Gyu-bon, Shin, Hyejung, Shin, Eun ji, Lee, Kang-in, Kim, Seokjoong, Lee, Jae Young, Song, Jihwan
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8770473/
https://www.ncbi.nlm.nih.gov/pubmed/35046408
http://dx.doi.org/10.1038/s41536-021-00198-0