Zinc Finger Nucleases (ZFNs) represent powerful tools for genome engineering in human cells. Yet, the full potential of this technology requires solving the challenge of delivering the required machinery 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 target cells. IDLV-mediated delivery supported high rates of gene editing in different cell types. IDLVs also mediated site-specific gene addition via a process that required ZFN cleavage and homologous template DNA, thus permitting the design of a platform that can target the insertion of transgenes into a pre-determined genomic site. Site-specific gene addition was observed at previously unattained levels in a panel of human cells, including hematopoietic and embryonic stem cells, allowing the rapid isolation of clonogenic cells with the desired genetic modification. These results open new avenues for experimental biology, biotechnology and medicine.</P>
"Blood Cells, Molecules and Diseases" – Elsevier
Published: Mar 1, 2008
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