ArticleScience translational medicine2021
Development of β-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease.
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Article in Science translational medicine, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 102 papers.
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102citing papers in PubMed
14.6field-weighted citation impact, top 1% of its field
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Who cites it
102 citing papers in PubMed, 152 citations in OpenAlex.
- Lentiviral gene therapy with reduced-intensity conditioning for sickle cell disease: a phase 1/2 trial.Nature medicine · 2025Trial
- Reverse genetics in humanized mice reveals CARD8-mediated pyroptosis causing pancytopenia in human DPP9 deficiency.The Journal of clinical investigation · 2026Article
- AAV vector production in suspension cells using PEI transfection and sodium butyrate with orthogonal assessment of function and quality.Molecular therapy. Advances · 2026Article
- In-depth characterization of stem cell potency and genotoxicity for clinical-scaleMolecular therapy. Advances · 2026Article
- Recent Advances in CRISPR/Cas Technologies for Biological Discovery, Therapeutics, and Diagnostics.Biomolecules · 2026Review
- Delivery Systems for Therapeutic Genome Editing: Challenges, Innovations, and Future Perspectives.MedComm · 2026Review
- Reverse genetics in humanized mice reveals CARD8-mediated pyroptosis causing pancytopenia in human DPP9 deficiency.bioRxiv : the preprint server for biology · 2026Article
- Illuminating "the dark side of the HBB": Visualizing the full spectrum of Cas9 outcomes.Molecular therapy. Nucleic acids · 2026Article
- Combinatorial base editing couples disease correction with lineage amplification in hematopoietic stem and progenitor cells.bioRxiv : the preprint server for biology · 2026Article
- DNA-PKcs inhibitor AZD7648 reveals sgRNA cross-contaminants and enhanced sensitivity of genome engineering off-target activity in HSPCs.Nucleic acids research · 2026Article
- Engineered hematopoietic stem cells give rise to therapeutic antibody secreting B cells.Research square · 2026Article
- CRISPR-Cas9 Therapeutics in Early Clinical Development: Delivery and Molecular Diagnostics.Cells · 2026Review
- Complex HBB gene editing outcomes revealed by a fluorescent reporter cell model.Molecular therapy. Nucleic acids · 2026Article
- High efficiency CRISPR knock-in demonstrates that TCF1 is insufficient to reverse T cell exhaustion.Nature communications · 2026Article
- CRISPR and Fanzor in sickle cell disease: current progress and future prospects.Frontiers in genome editing · 2026Review
- Gene therapy for hemoglobinopathies: Clinical trial results and biology of hematopoietic stem cell and the bone marrow niche.Cell reports. Medicine · 2025Review
- CRISPR-based therapeutic genome editing for inherited blood disorders.Nature reviews. Drug discovery · 2025Review
- Circular single stranded DNA potentiates non-viral gene insertion in hematopoietic stem and progenitor cells.Nature communications · 2025Article
- Review
- Genetic Engineering in Hematopoietic Stem Cells for β-Hemoglobinopathies Treatment: Advances, Challenges, and Clinical Translation.International journal of hematology-oncology and stem cell research · 2025Review
42 more citing papers are in PubMed but not listed here.
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Authors and funding
23 authors at 8 institutions in 3 countries.
Annalisa LattanziDepartment of Pediatrics, Stanford University, Stanford, CA 94305, USA.ORCID 0000-0002-3953-9172
Joab CamarenaDepartment of Pediatrics, Stanford University, Stanford, CA 94305, USA.ORCID 0000-0002-3102-5820
Premanjali LahiriLaboratory for Cell and Gene Medicine, Stanford University, Stanford, CA 94304, USA.ORCID 0000-0001-6885-0448
Helen SegalLaboratory for Cell and Gene Medicine, Stanford University, Stanford, CA 94304, USA.
Waracharee SrifaDepartment of Pediatrics, Stanford University, Stanford, CA 94305, USA.
Christopher A VakulskasIntegrated DNA Technologies Inc., Coralville, IA 52241, USA.
Richard L FrockDepartment of Radiation Oncology, Stanford University, Stanford, CA 94305, USA.ORCID 0000-0001-6963-4931
Josefin KenrickDepartment of Radiation Oncology, Stanford University, Stanford, CA 94305, USA.ORCID 0000-0002-0110-5192
Ciaran LeeAPC Microbiome Ireland, University College Cork, T12 YN60 Cork, Ireland.ORCID 0000-0002-0789-9149
Narae TalbottLaboratory for Cell and Gene Medicine, Stanford University, Stanford, CA 94304, USA.
Jason SkowronskiLaboratory for Cell and Gene Medicine, Stanford University, Stanford, CA 94304, USA.ORCID 0000-0002-2241-1148
M Kyle CromerDepartment of Pediatrics, Stanford University, Stanford, CA 94305, USA.
Carsten T CharlesworthDepartment of Pediatrics, Stanford University, Stanford, CA 94305, USA.ORCID 0000-0002-8588-6204
Rasmus O BakDepartment of Biomedicine, Aarhus University, DK-8000 Aarhus, Denmark.
Sruthi MantriDepartment of Pediatrics, Stanford University, Stanford, CA 94305, USA.ORCID 0000-0002-3423-8469
Gang BaoDepartment of Bioengineering, Rice University, Houston, TX 77006, USA.ORCID 0000-0001-5501-554X
David DiGiustoLaboratory for Cell and Gene Medicine, Stanford University, Stanford, CA 94304, USA.
John TisdaleMolecular and Clinical Hematology Branch, NHLBI, Bethesda, MD 20814, USA.ORCID 0000-0002-8535-9439
J Fraser WrightDepartment of Pediatrics, Stanford University, Stanford, CA 94305, USA.ORCID 0000-0001-9635-1750
Neehar BhatiaLaboratory for Cell and Gene Medicine, Stanford University, Stanford, CA 94304, USA.ORCID 0000-0002-9829-1453
Maria Grazia RoncaroloDepartment of Pediatrics, Stanford University, Stanford, CA 94305, USA.
Daniel P DeverDepartment of Pediatrics, Stanford University, Stanford, CA 94305, USA. dpdever@gmail.com mporteus@stanford.edu.ORCID 0000-0001-5966-4803
Matthew H PorteusDepartment of Pediatrics, Stanford University, Stanford, CA 94305, USA. dpdever@gmail.com mporteus@stanford.edu.ORCID 0000-0002-3850-4648
Stanford University · USCalifornia Institute for Regenerative Medicine · USStanford Medicine · USAarhus University · DKIntegrated DNA Technologies (United States) · USMaryland Oncology Hematology · USRice University · USUniversity College Cork · IE
Funding
HBB gene-editing for treating sickle cell diseaseR01HL152314 · NHLBI · RICE UNIVERSITY · PI BAO, GANG · 2020 to 2023
$2.5MHomologous Recombination Mediated Gene Correction for the HemoglobinopathiesR01HL135607 · NHLBI · STANFORD UNIVERSITY · PI PORTEUS, MATTHEW H · 2018 to 2021
$1.6MNHLBI NIH HHS R01 HL135607NHLBI NIH HHS R01 HL152314
Abstract
Sickle cell disease (SCD) is the most common serious monogenic disease with 300,000 births annually worldwide. SCD is an autosomal recessive disease resulting from a single point mutation in codon six of the β-globin gene (
Indexed as
Anemia, Sickle CellHeterocyclic CompoundsAnimalsbeta-GlobinsCRISPR-Cas SystemsGene EditingHematopoietic Stem Cell MobilizationHematopoietic Stem CellsHumansMiceReproducibility of Resultsbeta-GlobinsHeterocyclic Compounds
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.