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

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

Abstract 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 lowerin...

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Bibliographic Details
Main Authors: Hyun Jung Park, Areum Han, Ji Yeon Kim, Jiwoo Choi, Hee Sook Bae, Gyu-bon Cho, Hyejung Shin, Eun ji Shin, Kang-in Lee, Seokjoong Kim, Jae Young Lee, Jihwan Song
Format: Article
Language:English
Published: Nature Portfolio 2022-01-01
Series:npj Regenerative Medicine
Online Access:https://doi.org/10.1038/s41536-021-00198-0

Internet

https://doi.org/10.1038/s41536-021-00198-0

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