CRISPR‐based therapeutic and modelling approaches in Huntington's disease: Progress, challenges and future directions
Huntington’s disease (HD) is an autosomal dominant neurodegenerative disorder caused by CAG‐repeat expansion in exon 1 of the huntingtin gene ( HTT ). Mutant huntingtin accumulation and somatic repeat expansion contribute to neuronal dysfunction, making HD a compelling target for CRISPR‐based intervention....
K. Zhakipbekov, Surayyo Eshkabilova, Z. Raximov et al.
· Clinical and Translational D... · 0 citations