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PAM-altering SNP-based allele-specific CRISPR-Cas9 therapeutic strategies for Huntington’s disease

Huntington’s disease (HD) is caused by an expanded CAG repeat in huntingtin (HTT). Since HD is dominant and loss of HTT leads to neurological abnormalities, safe therapeutic strategies require selective inactivation of mutant HTT. Previously, we proposed a concept of CRISPR-Cas9 using mutant-specifi...

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Detalles Bibliográficos
Autores principales:	Shin, Jun Wan, Hong, Eun Pyo, Park, Seri S., Choi, Doo Eun, Zeng, Sophia, Chen, Richard Z., Lee, Jong-Min
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2022
Materias:	Original Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9450073/ https://www.ncbi.nlm.nih.gov/pubmed/36092363 http://dx.doi.org/10.1016/j.omtm.2022.08.005

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