Ontology highlight
ABSTRACT:
SUBMITTER: De Ravin SS
PROVIDER: S-EPMC4824656 | biostudies-literature | 2016 Apr
REPOSITORIES: biostudies-literature
Nature biotechnology 20160307 4
Gene therapy with genetically modified human CD34(+) hematopoietic stem and progenitor cells (HSPCs) may be safer using targeted integration (TI) of transgenes into a genomic 'safe harbor' site rather than random viral integration. We demonstrate that temporally optimized delivery of zinc finger nuclease mRNA via electroporation and adeno-associated virus (AAV) 6 delivery of donor constructs in human HSPCs approaches clinically relevant levels of TI into the AAVS1 safe harbor locus. Up to 58% Ve ...[more]