Homology-driven genome editing in hematopoietic stem and progenitor cells using ZFN mRNA and AAV6 donors
Open accessAbstract
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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It states that AAV6 donor templates with homology arms efficiently deliver donor DNA into sensitive primary immune cells such as HSPCs and T cells.
“AAV6 homologous donor DNA templates are an efficient way to deliver donor templates for sensitive primary immune cells, including HSPCs and T-Cells15,35–40.”
From A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human hematopoietic stem and progenitor cells (2018) · cites this paper as [35]
The Results section ('HDR-mediated editing in HSPCs by ZFN mRNA and AAV6 donors'; page unavailable) identifies AAV6 as an effective vehicle for delivering homologous donor templates to CD34+ HSPCs. The paper also reports T cells arising after edited HSPCs were transplanted, but does not establish efficient donor-template delivery directly into T cells. That extension is not supported by this source. (LLM, conf=0.88)
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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
- Venue
- Nature Biotechnology
- Published
- 2015-11-09
- Volume
- 33
- Issue
- 12
- Pages
- 1256-1263
- ISSN
- 0733-222X (ISSN-L)
- 1087-0156
- 1546-1696
- 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
About these checks
Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.
Statistics and arithmetic are recomputed, not judged.
Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.
What counts as a claim ›How we measure confidence ›How we check this ›
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