Demonstrates gene repair efficacy in hematopoietic stem cells, suggesting wider applications for genetic disorders.
mice resulted in efficient engraftment and production of functional mature human myeloid and lymphoid cells for up to 5 months. Whole-exome sequencing detected no indels outside of the CYBB gene after gene correction. CRISPR-mediated gene editing of HSPCs may be applicable to other CGD mutations and other monogenic disorders of the hematopoietic system.
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Ravin et al. (2017) studied this question.
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