An RNA-targeting CRISPR–Cas13d system alleviates disease-related phenotypes in Huntington’s disease models

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 CAG(EX) RNA-targeting...

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Detalles Bibliográficos
Autores principales: 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.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group US 2022
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9829537/
https://www.ncbi.nlm.nih.gov/pubmed/36510111
http://dx.doi.org/10.1038/s41593-022-01207-1

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