ArticleCell stem cell2014
Efficient ablation of genes in human hematopoietic stem and effector cells using CRISPR/Cas9.
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Article in Cell stem cell, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 271 papers.
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271citing papers in PubMed
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271 citing papers in PubMed, 475 citations in OpenAlex.
- Trial
- Safety and feasibility of CRISPR-edited T cells in patients with refractory non-small-cell lung cancer.Nature medicine · 2020Trial
- CRISPR-Cas9 genome editing: technological advances, delivery strategies and precision engineering of primary cells for therapeutic application in genetic disorders and diabetes mellitus.Molecular biology reports · 2026Review
- Engineering hematopoietic stem and progenitor cells to generate red blood cells as viral traps against HIV-1.Molecular therapy. Advances · 2026Article
- CCR5 as a Therapeutic Target in HIV Disease: From CRISPR/Cas9 Gene Editing to Maraviroc-Mediated Inhibition.Viruses · 2026Review
- Nuclear RSK1 mediates interferon-γ-induced proinflammatory activation in human primary macrophages and humanized mice.Science advances · 2026Article
- Engineered lipid nanoparticles for in vivo and durable editing of haematopoietic stem cells within humanized mice.Nature biomedical engineering · 2026Article
- CRISPR-Cas9: Genome Engineering and Future Vaccine Applications.Molecular biotechnology · 2026Review
- Precise CRISPR-Mediated Editing of theInternational journal of molecular sciences · 2026Article
- A novel minimally humanized mouse model of aniridia for preclinical evaluation of CRISPR gene editing strategies.Frontiers in genome editing · 2026Article
- A unified genetic perturbation language for human cellular programming.bioRxiv : the preprint server for biology · 2025Article
- Generation and functional analysis of melanoma antigen-specific CD8+ T cells derived from S/MAR vector-transfected human induced pluripotent stem cells.International journal of cancer · 2025Article
- Selective HLA knockdown and PD-L1 expression prevent allogeneic CAR-NK cell rejection and enhance safety and anti-tumor responses in xenograft mice.Nature communications · 2025Article
- Hypoimmune stem cells and islets: hype or a true breakthrough in diabetes treatment?Cellular & molecular biology letters · 2025Review
- Assessment the Efficacy of the CRISPR System for Inducing Mutations in the AIMP2 Gene to Create a Cell Line Model of HLD17 Disease.Molecular biotechnology · 2025Article
- Selective agonists of KIR and NKG2A to evade missing self response of natural killer cells.Scientific reports · 2025Article
- Off-target interactions in the CRISPR-Cas9 Machinery: mechanisms and outcomes.Biochemistry and biophysics reports · 2025Review
- Ligand-modified rAAV6 vectors with nanoblades allow high-level gene knockin in HSPCs without compromising cell survival.Molecular therapy. Nucleic acids · 2025Article
- New hope and promise with CRISPR-Cas9 technology for the treatment of HIV.Functional & integrative genomics · 2025Review
- Scalable intracellular delivery via microfluidic vortex shedding enhances the function of chimeric antigen receptor T-cells.Scientific reports · 2025Article
211 more citing papers are in PubMed but not listed here.
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Authors and funding
17 authors at 7 institutions in 2 countries.
Pankaj K MandalDepartment of Stem Cell and Regenerative Biology, Harvard University, Cambridge, MA 02138, USA; Program in Cellular and Molecular Medicine, Division of Hematology/Oncology, Boston Children's Hospital, Boston, MA 02116, USA.
Leonardo M R FerreiraDepartment of Stem Cell and Regenerative Biology, Harvard University, Cambridge, MA 02138, USA; Department of Molecular and Cellular Biology, Harvard University, Cambridge, MA 02138, USA.
Ryan CollinsMolecular Neurogenetics Unit, Psychiatric and Neurodevelopmental Genetics Unit, Center for Human Genetic Research, Massachusetts General Hospital, Boston, MA 02114, USA.
Torsten B MeissnerDepartment of Stem Cell and Regenerative Biology, Harvard University, Cambridge, MA 02138, USA.
Christian L BoutwellRagon Institute of MGH, MIT and Harvard, Cambridge, MA 02139, USA.
Max FriesenDepartment of Stem Cell and Regenerative Biology, Harvard University, Cambridge, MA 02138, USA.
Vladimir VrbanacRagon Institute of MGH, MIT and Harvard, Cambridge, MA 02139, USA; Center for Immunology and Inflammatory Diseases, Massachusetts General Hospital and Harvard Medical School, Charlestown, MA 02129, USA.
Brian S GarrisonDepartment of Stem Cell and Regenerative Biology, Harvard University, Cambridge, MA 02138, USA; Program in Cellular and Molecular Medicine, Division of Hematology/Oncology, Boston Children's Hospital, Boston, MA 02116, USA; Department of Pediatrics, Harvard Medical School, Boston, MA 02115, USA.
Alexei StortchevoiMolecular Neurogenetics Unit, Psychiatric and Neurodevelopmental Genetics Unit, Center for Human Genetic Research, Massachusetts General Hospital, Boston, MA 02114, USA.
David BryderInstitution for Experimental Medical Research, Immunology section, Lund University, 221 84, Lund, Sweden.
Kiran MusunuruDepartment of Stem Cell and Regenerative Biology, Harvard University, Cambridge, MA 02138, USA; Harvard Stem Cell Institute, Cambridge, MA 02138, USA; Division of Cardiovascular Medicine, Brigham and Women's Hospital, Boston, MA 02115, USA; Broad Institute, Cambridge, MA 02142, USA.
Harrison BrandMolecular Neurogenetics Unit, Psychiatric and Neurodevelopmental Genetics Unit, Center for Human Genetic Research, Massachusetts General Hospital, Boston, MA 02114, USA.
Andrew M TagerRagon Institute of MGH, MIT and Harvard, Cambridge, MA 02139, USA; Center for Immunology and Inflammatory Diseases, Massachusetts General Hospital and Harvard Medical School, Charlestown, MA 02129, USA.
Todd M AllenRagon Institute of MGH, MIT and Harvard, Cambridge, MA 02139, USA.
Michael E TalkowskiMolecular Neurogenetics Unit, Psychiatric and Neurodevelopmental Genetics Unit, Center for Human Genetic Research, Massachusetts General Hospital, Boston, MA 02114, USA; Broad Institute, Cambridge, MA 02142, USA; Department of Neurology, Harvard Medical School, Boston, MA 02115, USA.
Derrick J RossiDepartment of Stem Cell and Regenerative Biology, Harvard University, Cambridge, MA 02138, USA; Program in Cellular and Molecular Medicine, Division of Hematology/Oncology, Boston Children's Hospital, Boston, MA 02116, USA; Department of Pediatrics, Harvard Medical School, Boston, MA 02115, USA; Harvard Stem Cell Institute, Cambridge, MA 02138, USA. Electronic address: derrick.rossi@childrens.harvard.edu.
Chad A CowanDepartment of Stem Cell and Regenerative Biology, Harvard University, Cambridge, MA 02138, USA; Harvard Stem Cell Institute, Cambridge, MA 02138, USA; Broad Institute, Cambridge, MA 02142, USA; Center for Regenerative Medicine, Massachusetts General Hospital, Boston, MA 02114, USA. Electronic address: chad_cowan@harvard.edu.
Harvard University · USCenter for Human Genetics · USMassachusetts General Hospital · USRagon Institute of MGH, MIT and Harvard · USBoston Children's Hospital · USBrigham and Women's Hospital · USLund University · SE
Funding
The impact of HIV viral diversity and cellular immunity on HIV pathogenesisP30AI060354 · NIAID · HARVARD UNIVERSITY (MEDICAL SCHOOL) · PI RAJESH T GANDHI · 2004 to 2026
$94.4MCoordinating Center for Beta Cell Biology ConsortiumU01DK072473 · NIDDK · VANDERBILT UNIVERSITY · PI MAGNUSON, MARK A · 2005 to 2014
$61.0MVaccine Adjuvant/ Delivery Systems CoreP01AI104715 · NIAID · MASSACHUSETTS GENERAL HOSPITAL · PI ALLEN, TODD M · 2013 to 2017
$12.3MIsogenic Human Pluripotent Stem Cell-Based Models of Human Disease MutationsR01DK097768 · NIDDK · BROAD INSTITUTE, INC. · PI COWAN, CHAD ALBERT, ZHANG, FENG · 2012 to 2016
$11.0MIntegrating lipid genotyes and phenotypes in iPS derived hepatocytes/adipocytesU01HL107440 · NHLBI · MASSACHUSETTS GENERAL HOSPITAL · PI COWAN, CHAD ALBERT, DEO, RAHUL CHANDRAKANT · 2011 to 2015
$9.0MHuman Pluripotent Stem Cell and Progenitor Models of Cardiac and Blood DiseasesU01HL100408 · NHLBI · MASSACHUSETTS GENERAL HOSPITAL · PI COWAN, CHAD ALBERT, MELTON, DOUGLAS A · 2009 to 2015
$8.9MReprogramming committed blood cells to a hematopoietic stem cell fateR01HL107630 · NHLBI · IMMUNE DISEASE INSTITUTE, INC. · PI ROSSI, DERRICK J. · 2011 to 2015
$2.5MComplex Genetic Architecture of Chromosomal Aberrations in AutismR00MH095867 · NIMH · MASSACHUSETTS GENERAL HOSPITAL · PI TALKOWSKI, MICHAEL E · 2014 to 2016
$747kComplex Genetic Architecture of Chromosomal Aberrations in AutismK99MH095867 · NIMH · MASSACHUSETTS GENERAL HOSPITAL · PI TALKOWSKI, MICHAEL E · 2012 to 2013
$186kNHLBI NIH HHS R01 HL107630NHLBI NIH HHS R01HL107630NHLBI NIH HHS U01 HL100408NHLBI NIH HHS U01HL100408NHLBI NIH HHS U01 HL107440NIAID NIH HHS P01 AI104715NIAID NIH HHS P01-AI104715NIAID NIH HHS P30 AI060354NIDDK NIH HHS R01 DK097768NIDDK NIH HHS R01DK097768NIDDK NIH HHS U01 DK072473NIDDK NIH HHS U01DK072473NIMH NIH HHS K99 MH095867NIMH NIH HHS MH095867NIMH NIH HHS R00 MH095867
Abstract
Genome editing via CRISPR/Cas9 has rapidly become the tool of choice by virtue of its efficacy and ease of use. However, CRISPR/Cas9-mediated genome editing in clinically relevant human somatic cells remains untested. Here, we report CRISPR/Cas9 targeting of two clinically relevant genes, B2M and CCR5, in primary human CD4+ T cells and CD34+ hematopoietic stem and progenitor cells (HSPCs). Use of single RNA guides led to highly efficient mutagenesis in HSPCs but not in T cells. A dual guide approach improved gene deletion efficacy in both cell types. HSPCs that had undergone genome editing with CRISPR/Cas9 retained multilineage potential. We examined predicted on- and off-target mutations via target capture sequencing in HSPCs and observed low levels of off-target mutagenesis at only one site. These results demonstrate that CRISPR/Cas9 can efficiently ablate genes in HSPCs with minimal off-target mutagenesis, which could have broad applicability for hematopoietic cell-based therapy.
Indexed as
Gene DeletionAnimalsAntigens, CD34Cell LineageCells, CulturedClustered Regularly Interspaced Short Palindromic RepeatsCRISPR-Associated ProteinsGene TargetingGenetic LociGenome, HumanHematopoietic Stem CellsHigh-Throughput Nucleotide SequencingHumansMiceReceptors, CCR5RNA EditingAntigens, CD34CCR5 protein, humanCRISPR-Associated ProteinsReceptors, CCR5RNA, Guide, CRISPR-Cas Systems
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