Application of a Spacer-nick Gene-targeting Approach to Repair Disease-causing Mutations with Increased Safety

The CRISPR/Cas9 system is a powerful tool for gene repair that holds great potential for gene therapy to cure monogenic diseases. Despite intensive improvement, the safety of this system remains a major clinical concern. In contrast to Cas9 nuclease, Cas9 nickases with a pair of short-distance (38–6...

Descripción completa

Detalles Bibliográficos
Autores principales: Tran, Ngoc Tung, Lebedin, Mikhail, Danner, Eric, Kühn, Ralf, Rajewsky, Klaus, Chu, Van Trung
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Bio-Protocol 2023
Materias:
Methods Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10127048/
https://www.ncbi.nlm.nih.gov/pubmed/37113334
http://dx.doi.org/10.21769/BioProtoc.4661

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10127048/
https://www.ncbi.nlm.nih.gov/pubmed/37113334
http://dx.doi.org/10.21769/BioProtoc.4661

Ejemplares similares