Ontology highlight
ABSTRACT:
SUBMITTER: Paul B
PROVIDER: S-EPMC6054698 | biostudies-literature | 2018 Jun
REPOSITORIES: biostudies-literature
Paul Biswajit B Ibarra Guillermo S Romano GSR Hubbard Nicholas N Einhaus Teresa T Astrakhan Alexander A Rawlings David J DJ Kiem Hans-Peter HP Peterson Christopher W CW
Molecular therapy. Methods & clinical development 20180405
Targeted gene therapy strategies utilizing homology-driven repair (HDR) allow for greater control over transgene integration site, copy number, and expression-significant advantages over traditional vector-mediated gene therapy with random genome integration. However, the relatively low efficiency of HDR-based strategies limits their clinical application. Here, we used HDR to knock in a mutant dihydrofolate reductase (mDHFR) selection gene at the gene-edited CCR5 locus in primary human CD4<sup>+ ...[more]