Genomic Engineering in Human Hematopoietic Stem Cells: Hype or Hope?

ABSTRACT: Many gene editing techniques are developed and tested, yet, most of these are optimized for transformed cell lines, which differ from their primary cell counterparts in terms of transfectability, cell death propensity, differentiation capability, and chromatin accessibility to gene editing tools. Researchers are working to overcome the challenges associated with gene editing of primary cells, namely, at the level of improving the gene editing tool components, e.g., the use of modified single guide RNAs, more efficient delivery of Cas9 and RNA in the ribonucleoprotein of these cells. Despite these efforts, the low efficiency of proper gene editing in true primary cells is an obstacle that needs to be overcome in order to generate sufficiently high numbers of corrected cells for therapeutic

SUBMITTER: Klaver-Flores S 

PROVIDER: S-EPMC8525357 | biostudies-literature | 2020

REPOSITORIES: biostudies-literature