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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Бібліографічні деталі
Автори: 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
Формат: Journal article
Мова:English
Опубліковано: Springer Nature 2019