CRISPR-Based Genome-Editing Tools for Huntington’s Disease Research and Therapy

摘要

Huntington’s disease(HD)is an autosomal dominantly-inherited neurodegenerative disease,which is caused by CAG trinucleotide expansion in exon 1 of the Huntingtin(HTT)gene.Although HD is a rare disease,its monogenic nature makes it an ideal model in which to understand pathogenic mechanisms and to develop therapeutic strategies for neurodegenerative diseases.Clustered regularly-interspaced short palindromic repeats(CRISPR)is the latest technology for genome editing.Being simple to use and highly efficient,CRISPR-based genome-editing tools are rapidly gaining popularity in biomedical research and opening up new avenues for disease treatment.Here,we review the development of CRISPR-based genome-editing tools and their applications in HD research to offer a translational perspective on advancing the genome-editing technology to HD treatment.