A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human haematopoietic stem and progenitor cells

Translation of the CRISPR/Cas9 system to human therapeutics holds high promise. Specificity remains a concern, however, especially when modifying stem cell populations. We show that existing rationally-engineered Cas9 high fidelity variants have reduced on-target activity using the therapeutically r...

Descripción completa

Detalles Bibliográficos
Autores principales: Vakulskas, Christopher A., Dever, Daniel P., Rettig, Garrett R., Turk, Rolf, Jacobi, Ashley M., Collingwood, Michael A., Bode, Nicole M., McNeill, Matthew S., Yan, Shuqi, Camarena, Joab, Lee, Ciaran M., Park, So Hyun, Wiebking, Volker, Bak, Rasmus O., Gomez-Ospina, Natalia, Pavel-Dinu, Mara, Sun, Wenchao, Bao, Gang, Porteus, Matthew H., Behlke, Mark A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2018
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6107069/
https://www.ncbi.nlm.nih.gov/pubmed/30082871
http://dx.doi.org/10.1038/s41591-018-0137-0

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6107069/
https://www.ncbi.nlm.nih.gov/pubmed/30082871
http://dx.doi.org/10.1038/s41591-018-0137-0

Ejemplares similares