Journal
NATURE BIOTECHNOLOGY
Volume 25, Issue 11, Pages 1298-1306Publisher
NATURE PUBLISHING GROUP
DOI: 10.1038/nbt1353
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Funding
- NHLBI NIH HHS [2 P01 HL053750-11] Funding Source: Medline
- Telethon [TGT06S01] Funding Source: Medline
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Achieving the full potential of zinc-finger nucleases (ZFNs) for genome engineering in human cells requires their efficient delivery to the relevant cell types. Here we exploited the infectivity of integrase-defective lentiviral vectors (IDLV) to express ZFNs and provide the template DNA for gene correction in different cell types. IDLV-mediated delivery supported high rates (13-39%) of editing at the IL-2 receptor common gamma-chain gene (IL2RG) across different cell types. IDLVs also mediated sitespecific gene addition by a process that required ZFN cleavage and homologous template DNA, thus establishing a platform that can target the insertion of transgenes into a predetermined genomic site. Using IDLV delivery and ZFNs targeting distinct loci, we observed high levels of gene addition (up to 50%)in a panel of human cell lines, as well as human embryonic stem cells (5%), allowing rapid, selection-free isolation of clonogenic cells with the desired genetic modification.
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