RNA-Targeting CRISPR/CasRx system relieves disease symptoms in Huntington’s disease models

RNA-Targeting CRISPR/CasRx system relieves disease symptoms in Huntington’s disease models

Abstract Background HD is a devastating neurodegenerative disorder caused by the expansion of CAG repeats in the HTT. Silencing the expression of mutated proteins is a therapeutic direction to rescue HD patients, and recent advances in gene editing technology such as CRISPR/CasRx have opened up new...

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Main Authors: Yingqi Lin, Caijuan Li, Yizhi Chen, Jiale Gao, Jiawei Li, Chunhui Huang, Zhaoming Liu, Wei Wang, Xiao Zheng, Xichen Song, Jianhao Wu, Jiaxi Wu, Oscar Junhong Luo, Zhuchi Tu, Shihua Li, Xiao-Jiang Li, Liangxue Lai, Sen Yan
Format: Article
Language:English
Published: BMC 2025-01-01
Series:Molecular Neurodegeneration
Subjects:
CRISPR/CasRx
Huntington's disease
HTT
HD-KI pig
Online Access:https://doi.org/10.1186/s13024-024-00794-w
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https://doi.org/10.1186/s13024-024-00794-w

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