Precise CRISPR-Cas–mediated gene repair with minimal off-target and unintended on-target mutations in human hematopoietic stem cells
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DOI: 10.1126/sciadv.abm9106
Abstract: While CRISPR-Cas9 is key for the development of gene therapy, its potential off-target mutations are still a major concern. Here, we establish a “spacer-nick” gene correction approach that combines the Cas9D10A nickase with a pair… read more here.
Keywords: target mutations; human hematopoietic; hematopoietic stem; spacer nick ... See more keywords