Effective CRISPR/Cas9-mediated correction of a Fanconi anemia defect by error-prone end joining or templated repair

Fanconi anemia (FA) is a cancer predisposition syndrome characterized by congenital abnormalities, bone marrow failure, and hypersensitivity to aldehydes and crosslinking agents. For FA patients, gene editing holds promise for therapeutic applications aimed at functionally restoring mutated genes in...

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Main Authors: Van De Vrugt, HJ, Harmsen, T, Riepsaame, J, Alexantya, G, Van Mil, SE, De Vries, Y, Bin Ali, R, Huijbers, IJ, Dorsman, JC, Wolthuis, RMF, Riele, H
格式: Journal article
語言:English
出版: Springer Nature 2019