Cited by 261CRISPR and Genetic EngineeringVirus-based gene therapy researchRNA regulation and disease

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Homology-driven genome editing in hematopoietic stem and progenitor cells using ZFN mRNA and AAV6 donors

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Wang, Jianbin, Exline, Colin M, DeClercq, Joshua J, Llewellyn, G Nicholas, Hayward, Samuel B, Li, Patrick Wai-Lun, Shivak, David A, Surosky, Richard T, Gregory, Philip D, Holmes, Michael C, Cannon, Paula M·2015·Nature Biotechnology

DOI 10.1038/nbt.3408PMC PMC4842001

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Abstract

Genome editing with targeted nucleases and DNA donor templates homologous to the break site has proven challenging in human hematopoietic stem and progenitor cells (HSPCs), and particularly in the most primitive, long-term repopulating cell population. Here we report that combining electroporation of zinc finger nuclease (ZFN) mRNA with donor template delivery by adeno-associated virus (AAV) serotype 6 vectors directs efficient genome editing in HSPCs, achieving site-specific insertion of a GFP cassette at the CCR5 and AAVS1 loci in mobilized peripheral blood CD34+ HSPCs at mean frequencies of 17% and 26%, respectively, and in fetal liver HSPCs at 19% and 43%, respectively. Notably, this approach modified the CD34+CD133+CD90+ cell population, a minor component of CD34+ cells that contains long-term repopulating hematopoietic stem cells (HSCs). Genome-edited HSPCs also engrafted in immune-deficient mice long-term, confirming that HSCs are targeted by this approach. Our results provide a strategy for more robust application of genome-editing technologies in HSPCs.

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Authors: Wang, J., Exline, C. M., DeClercq, J. J., Llewellyn, G. N., Hayward, S. B., Li, P. W.-L., Shivak, D. A., Surosky, R. T., Gregory, P. D., Holmes, M. C., & Cannon, P. M.
Affiliations: Sangamo BioSciences (United States) (US)
University of Southern California (US)
Corresponding author: Holmes, Michael C
Cannon, Paula M
ORCID: Hayward, Samuel B
Surosky, Richard T
Gregory, Philip D
Holmes, Michael C
Cannon, Paula M
Venue: Nature Biotechnology
Publisher: Springer Science and Business Media LLC
Published: 2015-11-09
Volume: 33
Issue: 12
Pages: 1256-1263
ISSN: 0733-222X (ISSN-L)
1087-0156
1546-1696
DOI: 10.1038/nbt.3408
PMC: PMC4842001
OpenAlex: W2230199130
Semantic Scholar: 53d1b136b5c5b110dfdadc143c98505fc8c15d6b
Cited by: 261
References: 68
Citations by year: 2026: 6 · 2025: 9 · 2024: 13 · 2023: 15 · 2022: 20 · 2021: 26 · 2020: 24 · 2019: 31 · 2018: 35 · 2017: 39 · 2016: 41 · 2015: 2
FWCI: 16.93
Type: journal article
Language: en
Open access: green
www.ncbi.nlm.nih.gov
Licence: www.springer.com/tdm (tdm)
Locations: Nature Biotechnology (publishedVersion)
PubMed (publishedVersion)
Europe PMC (PubMed Central) (acceptedVersion)
PubMed Central (acceptedVersion)
Funders: National Heart, Lung, and Blood Institute (P01HL073104, U19HL129902)
National Institute of Dental and Craniofacial Research (R01DE025167)
Topics: Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › CRISPR and Genetic Engineering
Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA regulation and disease
Keywords: hematopoietic stem and progenitor cells, homology-directed repair, zinc finger nucleases, AAV6 vector, mRNA electroporation, CCR5, AAVS1, long-term repopulating cells, fetal liver, peripheral blood stem cell mobilization, gene targeting

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Homology-driven genome editing in hematopoietic stem and progenitor cells using ZFN mRNA and AAV6 donors

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