Precise CRISPR-Cas–mediated gene repair with minimal off-target and unintended on-target mutations in human hematopoietic stem cells

While CRISPR-Cas9 is key for the development of gene therapy, its potential off-target mutations are still a major concern. Here, we establish a “spacer-nick” gene correction approach that combines the Cas9(D10A) nickase with a pair of PAM-out sgRNAs at a distance of 200 to 350 bp. In combination wi...

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Detalles Bibliográficos
Autores principales: Tran, Ngoc Tung, Danner, Eric, Li, Xun, Graf, Robin, Lebedin, Mikhail, de la Rosa, Kathrin, Kühn, Ralf, Rajewsky, Klaus, Chu, Van Trung
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Association for the Advancement of Science 2022
Materias:
Biomedicine and Life Sciences
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9166625/
https://www.ncbi.nlm.nih.gov/pubmed/35658035
http://dx.doi.org/10.1126/sciadv.abm9106

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