CRISPR/Cas9 genome editing in human hematopoietic stem cells.
Open accessAbstract
Genome editing via homologous recombination (HR) (gene targeting) in human hematopoietic stem cells (HSCs) has the power to reveal gene-function relationships and potentially transform curative hematological gene and cell therapies. However, there are no comprehensive and reproducible protocols for targeting HSCs for HR. Herein, we provide a detailed protocol for the production, enrichment, and in vitro and in vivo analyses of HR-targeted HSCs by combining CRISPR/Cas9 technology with the use of rAAV6 and flow cytometry. Using this protocol, researchers can introduce single-nucleotide changes into the genome or longer gene cassettes with the precision of genome editing. Along with our troubleshooting and optimization guidelines, researchers can use this protocol to streamline HSC genome editing at any locus of interest. The in vitro HSC-targeting protocol and analyses can be completed in 3 weeks, and the long-term in vivo HSC engraftment analyses in immunodeficient mice can be achieved in 16 weeks. This protocol enables manipulation of genes for investigation of gene functions during hematopoiesis, as well as for the correction of genetic mutations in HSC transplantation-based therapies for diseases such as sickle cell disease, β-thalassemia, and primary immunodeficiencies.
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CRISPR-Cas9 enables programmable targeting of indels at a chosen genomic site.
“The clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 nuclease system, a bacterial immune system that can cleave bacteriophage or plasmid DNA, enables programmable targeting of insertions or deletions (indels) at a specific genomic DNA site.28,29”
From CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia (2021) · cites this paper as [28]
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Self-citation
- Authors
- Bak, R. O., Dever, D. P., & Porteus, M. H.
- Affiliations
- Stanford Medicine (US)
- Corresponding author
- Porteus, Matthew H
- Venue
- Nature Protocols
- Published
- 2018-01-25
- Volume
- 13
- Issue
- 2
- Pages
- 358-376
- ISSN
- 1750-2799 (ISSN-L)
- 1754-2189
- PMC
- PMC5826598
- OpenAlex
- W2784423079
- Cited by
- 319
- References
- 57
- Citations by year
- 2026: 23 · 2025: 37 · 2024: 31 · 2023: 47 · 2022: 35 · 2021: 51 · 2020: 50 · 2019: 31 · 2018: 13
- FWCI
- 12.88
- Type
- journal article
- Language
- en
- Open access
- green
- www.ncbi.nlm.nih.gov
- Locations
- Nature Protocols (publishedVersion)
- PubMed (publishedVersion)
- Europe PMC (PubMed Central) (acceptedVersion)
- PubMed Central (acceptedVersion)
- Funders
- National Institutes of Health (5r01ai120766-02)
- National Institute of Allergy and Infectious Diseases (R01-AI120766, R01AI097320)
- 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
- Health Sciences › Medicine › Hematology › Hematopoietic Stem Cell Transplantation
- Keywords
- CRISPR/Cas9 genome editing, hematopoietic stem cells, homologous recombination, AAV6 vector, Cas9 ribonucleoprotein, HSC engraftment, hematopoietic stem and progenitor cells, sickle cell disease, beta-thalassemia, primary immunodeficiency, gene therapy
- MeSH terms
- Gene Editing, Dependovirus, Animals, Flow Cytometry, Hematopoiesis, Hematopoietic Stem Cells, Humans, Genetic Therapy, Hematopoietic Stem Cell Transplantation, Gene Targeting, Polymorphism, Single Nucleotide, Mice, Homologous Recombination, CRISPR-Cas Systems
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