Selection-free genome editing of the sickle mutation in human adult hematopoietic stem/progenitor cells
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Hematopoietic stem cells from patients with sickle cell disease can be edited by CRISPR/Cas9 and maintain the edits in vivo.
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It reports that ribonucleoprotein delivery gives a brief pulse of editing machinery, producing efficient editing with fewer off-target effects.
“RNP delivery results in an initially high level of the genome editing machinery followed by rapid decay, leading to a “fast on, fast off” strategy that results in highly efficient editing while minimizing OTEs13–19.”
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 [16]
The Discussion reports efficient editing in human HSPCs with minimal genic off-target activity and notes that RNP delivery has been associated with reduced off-target effects. But our copy does not establish an initial high level followed by rapid decay, or show that such a pulse causes fewer off-target effects. The Results also report substantial activity at one intergenic off-target site, so the off-target finding needs that qualification. (LLM, conf=0.86)
Experimental — accuracy not yet measuredIt notes that multiple reports have achieved efficient Glu6Val correction in SCD HSPCs, though off-target insertions or deletions can accompany correction.
“Accordingly, several reports have shown efficient Glu6Val correction in SCD-HSPCs, although correction efficiency can sometimes be linked with off-target INDELs in the same edited population15,16,45,46.”
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 [16]
The Results section on correcting SCD HSPCs reports up to 25% of alleles corrected to wild type. A separate Results section on editing CD34+ HSPCs reports off-target indels, including 44% at an intergenic site, but does not establish that those indels occurred in the same SCD-HSPC population whose mutation was corrected. The paper therefore supports efficient correction and an off-target concern, but not that specific linkage. (LLM, conf=0.83)
Experimental — accuracy not yet measuredIt cites prior work showing that CRISPR/Cas9 can correct disease-causing mutations in long-term engrafting HSPCs that persist in downstream lineages.
“Recent studies have shown that disease-causing genetic errors can be corrected using CRISPR/Cas9 in long-term engrafting HSPCs that are preserved in downstream effector lineages15,16,24,25.”
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 [16]
The Results sections report CRISPR/Cas9 correction of the sickle mutation in patient HSPCs and, separately, durable editing after HSPC engraftment in mice. But the engraftment experiment edited wild-type HSPCs to carry the sickle mutation, rather than testing corrected patient HSPCs in vivo. The paper therefore does not show that correction of a disease-causing mutation persisted in downstream lineages after long-term engraftment. (LLM, conf=0.91)
Experimental — accuracy not yet measuredMajor progress in delivering to and handling HPSCs has resulted in formidable efficiencies of mutation correction or mitigation that are expected to be curative.
“Major progress in delivering to 44 and handling HPSCs has resulted in formidable efficiencies of mutation correction or mitigation 18,45-47 that are expected to be curative.”
From The promise and challenge of therapeutic genome editing (2020) · cites this paper as [46]
The Results report up to 25% correction of sickle-cell alleles in patient HSPCs in vitro, and the Discussion says editing levels in long-term stem cells may be clinically relevant (pages not supplied). But the paper does not say these efficiencies are expected to be curative; it cautions that correction may lessen rather than eliminate SCD symptoms. (LLM, conf=0.88)
Experimental — accuracy not yet measured
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Wienert, B., Martyn, G. E., Funnell, A. P. W., Quinlan, K. G. R., & Crossley, M. (2018) · Trends in Genetics · doi:10.1016/j.tig.2018.09.004 · Cited by 115 · see citing works
Dever, D. P., & Porteus, M. H. (2017) · Current Opinion in Hematology · doi:10.1097/moh.0000000000000385 · Cited by 62 · see citing works
Cas9 protein delivery non-integrating lentiviral vectors for gene correction in sickle cell disease
Uchida, N., Drysdale, C. M., Nassehi, T., Gamer, J., Yapundich, M., DiNicola, J., Shibata, Y., Hinds, M., Gudmundsdottir, B., Haro-Mora, J. J., et al. (2021) · Molecular Therapy - Methods & Clinical Development · doi:10.1016/j.omtm.2021.02.022 · Cited by 55 · see citing works
Tran, N. T., Danner, E., Li, X., Graf, R., Lebedin, M., de la Rosa, K., Kühn, R., Rajewsky, K., & Chu, V. T. (2022) · Science Advances · doi:10.1126/sciadv.abm9106 · Cited by 49 · see citing works
Curative options for sickle cell disease: haploidentical stem cell transplantation or gene therapy?
Leonard, A., Tisdale, J., & Abraham, A. (2020) · British Journal of Haematology · doi:10.1111/bjh.16437 · Cited by 45 · see citing works
Technical considerations for the use of CRISPR/Cas9 in hematology research
Gundry, M. C., Dever, D. P., Yudovich, D., Bauer, D. E., Haas, S., Wilkinson, A. C., & Singbrant, S. (2017) · Experimental Hematology · doi:10.1016/j.exphem.2017.07.006 · Cited by 26 · see citing works
The Daunting Economics of Therapeutic Genome Editing
Wilson, R. C., & Carroll, D. (2019) · The CRISPR Journal · doi:10.1089/crispr.2019.0052 · Cited by 33 · see citing works
Self-citation
- Authors
- DeWitt, M. A., Magis, W., Bray, N. L., Wang, T., Berman, J. R., Urbinati, F., Heo, S.-J., Mitros, T., Muñoz, D. P., Boffelli, D., Kohn, D. B., Walters, M. C., Carroll, D., Martin, D. I. K., & Corn, J. E.
- Affiliations
- Innovative Genomics Institute (US)
- University of California, San Francisco (US)
- Bio-Rad (United States) (US)
- University of California, Los Angeles (US)
- University of California, Berkeley (US)
- University of Utah (US)
- Corresponding author
- Carroll, Dana
- Martin, David I. K.
- Corn, Jacob E.
- ORCID
- Venue
- Science Translational Medicine
- Published
- 2016-10-12
- Volume
- 8
- Issue
- 360
- Pages
- 360ra134-360ra134
- ISSN
- 1946-6234 (ISSN-L)
- 1946-6242
- PMC
- PMC5500303
- OpenAlex
- W2530601336
- Semantic Scholar
- 39df12b515c1f9195e6fde7fb84e9be2e553a49b
- Cited by
- 477
- References
- 57
- Citations by year
- 2026: 18 · 2025: 28 · 2024: 54 · 2023: 38 · 2022: 35 · 2021: 57 · 2020: 59 · 2019: 62 · 2018: 61 · 2017: 61 · 2016: 3 · 2015: 1
- FWCI
- 32.39
- Type
- journal article
- Language
- en
- Open access
- green
- www.ncbi.nlm.nih.gov
- Locations
- Science Translational Medicine (publishedVersion)
- PubMed (publishedVersion)
- PubMed Central (acceptedVersion)
- eScholarship (California Digital Library) (submittedVersion)
- Funders
- Li Ka Shing Foundation
- Siebel Scholars Fund at Berkeley
- NIH (R01 GM078571, 5R21AA022753, 1R56ES022377-01)
- Jordan Family Fund
- Doris Duke Charitable Foundation
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › CRISPR and Genetic Engineering
- Health Sciences › Medicine › Genetics › Hemoglobinopathies and Related Disorders
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Keywords
- sickle cell disease, hematopoietic stem and progenitor cells, genome editing, Cas9 ribonucleoprotein, beta-globin gene, hemoglobin S, ex vivo gene therapy, engraftment, erythroblasts, sickle cell disease treatment
- MeSH terms
- Gene Editing, Adult, Anemia, Sickle Cell, Animals, Cell Line, Hematopoietic Stem Cells, Hemoglobin, Sickle, Humans, Mutation, Mice, SCID, Mice, Inbred NOD, Mice, Knockout, Polymorphism, Single Nucleotide, Mice, Adult Stem Cells, Translational Research, Biomedical, CRISPR-Cas Systems, Heterografts
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