Article
A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human hematopoietic stem and progenitor cells.
Nature medicine - 1 Aug 2018
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
Abstract excerpt
Translation of the CRISPR-Cas9 system to human therapeutics holds high promise. However, specificity remains a concern especially when modifying stem cell populations. We show that existing rationally engineered Cas9 high-fidelity variants have reduced on-target activity when using the therapeutically relevant ribonucleoprotein (RNP) delivery method. Therefore, we devised an unbiased bacterial screen to isolate...
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