CRISPR-Based Genome-Editing Tools for Huntington’s Disease Research and Therapy
作者机构：Guangdong Key Laboratory of Non-human Primate ResearchGuangdong-Hongkong-Macao Institute of CNS RegenerationJinan UniversityGuangzhou 510632China
出 版 物：《Neuroscience Bulletin》 (神经科学通报（英文版）)
年 卷 期：2022年第38卷第11期
页 面：1397-1408页
核心收录：
学科分类：1002[医学-临床医学] 100204[医学-神经病学] 10[医学]
基 金：the National Key R&D Program of China(2021YFA0805200) the National Natural Science Foundation of China(31970954,81901289 and 31872779) the Guangdong Key Laboratory of Non-human Primate Research(2020B121201006)
主 题：Huntington’s disease CRISPR Animal models
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)*** 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 *** regularly-interspaced short palindromic repeats(CRISPR)is the latest technology for genome *** 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 ***,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.
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