Technical considerations for the use of CRISPR/Cas9 in hematology research
Open accessAbstract
The hematopoietic system is responsible for transporting oxygen and nutrients, fighting infections, and repairing tissue damage. Hematopoietic system dysfunction therefore causes a range of serious health consequences. Lifelong hematopoiesis is maintained by repopulating multipotent hematopoietic stem cells (HSCs) that replenish shorter-lived, mature blood cell types. A prokaryotic mechanism of immunity, the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/Cas9 nuclease system, has recently been “repurposed” to efficiently mutate mammalian genomes in a sequence-specific manner. The application of this genome-editing technology to hematology has afforded new approaches for functional genomics and even the prospect of “correcting” dysfunctional HSCs in the treatment of serious genetic hematological diseases. In this Perspective, we provide an overview of three recent CRISPR/Cas9 methods in hematology: gene disruption, gene targeting, and saturating mutagenesis. We also summarize the technical considerations and advice provided during the May 2017 International Society of Experimental Hematology New Investigator Committee webinar on the same topic.
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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 [14]
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References 44 · Cited by 26
- Authors
- Gundry, M. C., Dever, D. P., Yudovich, D., Bauer, D. E., Haas, S., Wilkinson, A. C., & Singbrant, S.
- Affiliations
- Baylor College of Medicine (US)
- Stanford University (US)
- Lund University (SE)
- Boston Children's Hospital (US)
- German Cancer Research Center (DE)
- Institute for Stem Cell Biology and Regenerative Medicine (US)
- Corresponding author
- Wilkinson, Adam C.
- ORCID
- Venue
- Experimental Hematology
- Publisher
- Elsevier BV
- Published
- 2017-10
- Volume
- 54
- Pages
- 4-11
- ISSN
- 0301-472X (ISSN-L)
- PMC
- PMC5603407
- OpenAlex
- W2742080296
- Semantic Scholar
- a0aded902e158d7700996ab9ac1815fc57455cbf
- Cited by
- 26
- References
- 44
- Citations by year
- 2026: 2 · 2025: 2 · 2024: 8 · 2022: 1 · 2021: 4 · 2020: 5 · 2019: 1 · 2018: 3
- FWCI
- 0.23
- Type
- journal article
- Language
- en
- Open access
- bronze
- doi.org
- Licence
- Locations
- Experimental Hematology (publishedVersion)
- PubMed (publishedVersion)
- Europe PMC (PubMed Central) (acceptedVersion)
- PubMed Central (acceptedVersion)
- MDC Repository (Max-Delbrueck-Center for Molecular Medicine) (submittedVersion)
- Lund University Publications (Lund University) (submittedVersion)
- Funders
- Baylor College of Medicine Medical Scientist Training Program
- Cancer Prevention & Research Institute of Texas (RP160283)
- National Institute of Diabetes and Digestive and Kidney Diseases–National Institutes of Health (K08DK093705, R03DK109232)
- National Heart, Lung, and Blood Institute (DP2OD022716)
- Burroughs Wellcome Fund
- American Society of Hematology
- Doris Duke Charitable Foundation
- Charles H. Hood Foundation
- Cooley's Anemia Foundation
- Bloodwise (UK) Visiting Fellowship (15050)
- National Institutes of Health (NIH, USA) National Center for Advancing Translational Science Clinical and Translational Science Award (UL1 TR001085)
- Swedish Foundation for Medical Research
- StemTherapy
- The Crafoordska Foundation
- Ake Wiberg Foundation
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › CRISPR and Genetic Engineering
- Health Sciences › Medicine › Hematology › Hematopoietic Stem Cell Transplantation
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Virus-based gene therapy research
- Keywords
- CRISPR/Cas9, hematopoietic stem cells, hematopoiesis, gene disruption, gene targeting, genome editing, functional genomics
- MeSH terms
- Gene Editing, RNA, Guide, CRISPR-Cas Systems, Animals, Genetic Vectors, Hematology, Hematopoiesis, Hematopoietic Stem Cells, Humans, Lentivirus, Mutagenesis, Genome, Hematopoietic Stem Cell Transplantation, Gene Targeting, Computational Biology, Multipotent Stem Cells, CRISPR-Cas Systems
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