Allele-specific silencing of the gain-of-function mutation in Huntington’s disease using CRISPR/Cas9

Dominant gain-of-function mechanisms in Huntington’s disease (HD) suggest that selective silencing of mutant HTT produces robust therapeutic benefits. Here, capitalizing on exonic protospacer adjacent motif–altering (PAM-altering) SNP (PAS), we developed an allele-specific CRISPR/Cas9 strategy to pe...

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Detalles Bibliográficos
Autores principales: Shin, Jun Wan, Hong, Eun Pyo, Park, Seri S., Choi, Doo Eun, Seong, Ihn Sik, Whittaker, Madelynn N., Kleinstiver, Benjamin P., Chen, Richard Z., Lee, Jong-Min
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society for Clinical Investigation 2022
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9675467/
https://www.ncbi.nlm.nih.gov/pubmed/36040815
http://dx.doi.org/10.1172/jci.insight.141042

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