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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...
Autores principales: | , , , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Nature Publishing Group UK
2022
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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 |