Article
Controlling genetic heterogeneity in gene-edited hematopoietic stem cells by single-cell expansion.
Cell stem cell - 6 Jul 2023
Becker Hans Jiro, Ishida Reiko, Wilkinson Adam C, Kimura Takaharu, Lee Michelle Sue Jann, Coban Cevayir, Ota Yasunori, Tanaka Yosuke, Roskamp Meike, Sano Tsubasa, Tojo Arinobu, Kent David G, Yamazaki Satoshi
Abstract excerpt
Gene editing using engineered nucleases frequently produces unintended genetic lesions in hematopoietic stem cells (HSCs). Gene-edited HSC cultures thus contain heterogeneous populations, the majority of which either do not carry the desired edit or harbor unwanted mutations. In consequence, transplanting edited HSCs carries the risks of suboptimal efficiency and of unwanted mutations in the graft. Here, we...
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