Cited by 557CRISPR and Genetic EngineeringAcute Myeloid Leukemia ResearchChromatin Remodeling and Cancer

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Generation of mouse models of myeloid malignancy with combinatorial genetic lesions using CRISPR-Cas9 genome editing

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Heckl, Dirk, Kowalczyk, Monika S, Yudovich, David, Belizaire, Roger, Puram, Rishi V, McConkey, Marie E, Thielke, Anne, Aster, Jon C, Regev, Aviv, Ebert, Benjamin L·2014·Nature Biotechnology

DOI 10.1038/nbt.2951PMC PMC4160386

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Abstract

Genome sequencing studies have shown that human malignancies often bear mutations in four or more driver genes, but it is difficult to recapitulate this degree of genetic complexity in mouse models using conventional breeding. Here we use the CRISPR-Cas9 system of genome editing to overcome this limitation. By delivering combinations of small guide RNAs (sgRNAs) and Cas9 with a lentiviral vector, we modified up to five genes in a single mouse hematopoietic stem cell (HSC), leading to clonal outgrowth and myeloid malignancy. We thereby generated models of acute myeloid leukemia (AML) with cooperating mutations in genes encoding epigenetic modifiers, transcription factors and mediators of cytokine signaling, recapitulating the combinations of mutations observed in patients. Our results suggest that lentivirus-delivered sgRNA:Cas9 genome editing should be useful to engineer a broad array of in vivo cancer models that better reflect the complexity of human disease.

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Authors: Heckl, D., Kowalczyk, M. S., Yudovich, D., Belizaire, R., Puram, R. V., McConkey, M. E., Thielke, A., Aster, J. C., Regev, A., & Ebert, B. L.
Affiliations: Brigham and Women's Hospital (US)
Broad Institute (US)
Corresponding author: Ebert, Benjamin L
ORCID: Heckl, Dirk
Kowalczyk, Monika S
Yudovich, David
Belizaire, Roger
McConkey, Marie E
Aster, Jon C
Regev, Aviv
Ebert, Benjamin L
Venue: Nature Biotechnology
Publisher: Springer Science and Business Media LLC
Published: 2014-06-22
Volume: 32
Issue: 9
Pages: 941-946
ISSN: 0733-222X (ISSN-L)
1087-0156
1546-1696
DOI: 10.1038/nbt.2951
PMC: PMC4160386
OpenAlex: W1575193685
Cited by: 555
References: 27
Citations by year: 2026: 14 · 2025: 29 · 2024: 28 · 2023: 58 · 2022: 58 · 2021: 58 · 2020: 61 · 2019: 51 · 2018: 54 · 2017: 61 · 2016: 38 · 2015: 39 · 2014: 5
FWCI: 19.53
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)
PubMed Central (acceptedVersion)
Digital Access to Scholarship at Harvard (DASH) (Harvard University) (submittedVersion, cc-by)
DSpace@MIT (Massachusetts Institute of Technology) (submittedVersion, cc-by-nc-sa)
Funders: National Heart, Lung, and Blood Institute (R01HL082945)
National Human Genome Research Institute (P50HG006193)
National Cancer Institute (P01CA108631, P01CA066996)
National Institute of General Medical Sciences (T32GM007753)
Topics: Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › CRISPR and Genetic Engineering
Health Sciences › Medicine › Hematology › Acute Myeloid Leukemia Research
Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Chromatin Remodeling and Cancer
Keywords: myeloid neoplasms, acute myeloid leukemia, mouse tumor model, CRISPR/Cas9 genome editing, lentiviral delivery, hematopoietic stem cells, clonal outgrowth, driver mutations, epigenetic regulators, transcription factors, cytokine signaling, cooperating mutations
MeSH terms: Animals, Disease Models, Animal, Bone Marrow Neoplasms, Mice, Clustered Regularly Interspaced Short Palindromic Repeats

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Generation of mouse models of myeloid malignancy with combinatorial genetic lesions using CRISPR-Cas9 genome editing

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