Article
An RNA-targeting CRISPR-Cas13d system alleviates disease-related phenotypes in Huntington's disease models.
Nature neuroscience - 1 Jan 2023
Morelli Kathryn H, Wu Qian, Gosztyla Maya L, Liu Hongshuai, Yao Minmin, Zhang Chuangchuang, Chen Jiaxu, Marina Ryan J, Lee Kari, Jones Krysten L, Huang Megan Y, Li Allison, Smith-Geater Charlene, Thompson Leslie M, Duan Wenzhen, Yeo Gene W
Abstract excerpt
Huntington's disease (HD) is a fatal, dominantly inherited neurodegenerative disorder caused by CAG trinucleotide expansion in exon 1 of the huntingtin (HTT) gene. Since the reduction of pathogenic mutant HTT messenger RNA is therapeutic, we developed a mutant allele-sensitive CAGEX RNA-targeting CRISPR-Cas13d system (Cas13d-CAGEX) that eliminates toxic CAGEX RNA in fibroblasts derived from patients with HD and...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.