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Article

Long-term multilineage engraftment of autologous genome-edited hematopoietic stem cells in nonhuman primates.

Blood - 19 May 2016

Peterson Christopher W, Wang Jianbin, Norman Krystin K, Norgaard Zachary K, Humbert Olivier, Tse Collette K, Yan Jenny J, Trimble Richard G, Shivak David A, Rebar Edward J, Gregory Philip D, Holmes Michael C, Kiem Hans-Peter

Abstract excerpt

Genome editing in hematopoietic stem and progenitor cells (HSPCs) is a promising novel technology for the treatment of many human diseases. Here, we evaluated whether the disruption of the C-C chemokine receptor 5 (CCR5) locus in pigtailed macaque HSPCs by zinc finger nucleases (ZFNs) was feasible. We show that macaque-specific CCR5 ZFNs efficiently induce CCR5 disruption at levels of up to 64% ex vivo, 40% in...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 26980728
DOI: 10.1182/blood-2015-09-672337
PMCID: PMC4874223

Genes

CCR5

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