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Targeted, homology-driven gene insertion in stem cells by ZFN-loaded ‘all-in-one’ lentiviral vectors
Targeted, homology-driven gene insertion in stem cells by ZFN-loaded ‘all-in-one’ lentiviral vectors | Building on previous work (Cai et al., 2014), HIV-derived lentiviral vectors are harnessed to insert user-defined genes into a safe locus in the genome of human stem cells. 
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Here, the scientists design non integrating lentiviral vectors (NILVs) "all in one" which contains ZFN that insert transgenes by a homology-driven mechanism into safe loci. This insertion mechanism is driven by time-restricted exposure of treated cells to ZFNs. They show targeted gene integration in human stem cells, including CD34+ hematopoietic progenitors and induced pluripotent stem cells (iPSCs). Notably, targeted insertions are identified in 89% of transduced iPSCs. Their findings demonstrate the applicability of nuclease-loaded ‘all-in-one’ NILVs for site-directed gene insertion in stem cell-based gene therapies.