Gene-ectomy: Gene Ablation with CRISPR/Cas9 in Human Hematopoietic Cells

摘要

CRISPR/Cas9 has recently been introduced as a gene editing tool and shows considerable promise. In this issue of Ceil Stem Cell, Mandal et al. (2014) show efficient CRISPR/Cas9-mediated ablation of the CCR5 and B2M genes in primary human hematopoietic cells, two editing strategies that are potentially translatable into clinical application. Experimental medicine through the use of cell and stem cell therapies has the potential to transform the way we treat specific diseases, in particular, the manipulation of the human hematopoietic system has clinically led the gene and cell therapy field. Gene-modified autologous stem and mature T cell products have now shown considerable clinical efficacy in the treatment of a number of monogenic diseases and acquired conditions. To date, gene delivery has relied on the use of integrating vectors that cannot be directed to a specific gene or locus and thus have the potential for semirandom genomic disruption.