ArticleNature2021
Base editing of haematopoietic stem cells rescues sickle cell disease in mice.
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Article in Nature, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 251 papers.
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251citing papers in PubMed
21.7field-weighted citation impact, top 1% of its field
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Who cites it
251 citing papers in PubMed, 360 citations in OpenAlex.
- Lentiviral gene therapy with reduced-intensity conditioning for sickle cell disease: a phase 1/2 trial.Nature medicine · 2025Trial
- Gene therapy for hereditary hematological disorders: From clinical breakthroughs to future horizons.Molecular therapy. Nucleic acids · 2026Review
- Translating base editors: a decade of progress from the bench to the clinic.Nature reviews. Drug discovery · 2026Review
- Red lines and green lights: Gene therapy for inherited erythroid disorders beyond the haemoglobinopathies.British journal of haematology · 2026Review
- MN•CS•IO System: Cross-Species Study for HBB Function in High-Altitude Hypoxia Adaptation.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2026Article
- 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
- Beyond Precision: A Multidimensional Framework for Selecting Genetic Medicine Platforms.Cells · 2026Review
- Base editing for precision therapeutics.Cell genomics · 2026Review
- Gene editing of hematopoietic stem cells: applications and advances.International journal of hematology · 2026Review
- Mechanistic machine learning for prediction of prime editing outcomes.Nature biotechnology · 2026Article
- UNCOVERseq enables sensitive and controlled gene editing off-target nomination across CRISPR-Cas modalities and systems.Nature communications · 2026Article
- In vivo base editing of Asgr1 reduces blood lipids in mice.Molecular therapy : the journal of the American Society of Gene Therapy · 2026Article
- Sickle Cell Disease: From Ancient Origins to Modern Breakthroughs in Gene Therapy.Biomedicines · 2026Review
- Delivery Systems for Therapeutic Genome Editing: Challenges, Innovations, and Future Perspectives.MedComm · 2026Review
- Restoration of the immune system with base editing and non-genotoxic conditioning in a Rag2 point-mutant mouse model.Molecular therapy : the journal of the American Society of Gene Therapy · 2026Article
- Gene Therapy for β-Haemoglobinopathies: From Molecular Correction to Curative Medicine.Biomedicines · 2026Review
- CRISPR-Cas9-mediated homology-directed repair rescues the induced bone marrow failure inMolecular therapy. Nucleic acids · 2026Article
- Review
- Therapeutic adenine base editor with minimized off-target effects.Protein & cell · 2026Article
- CRISPR Genome Editing and the Future of Leukaemia Immunotherapy.Health science reports · 2026Article
191 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
29 authors at 5 institutions in 1 country.
Gregory A Newby *Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA, USA.
Jonathan S Yen *Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA. jonathan.yen@stjude.org.ORCID http://orcid.org/0000-0002-9432-9450
Kaitly J Woodard *Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.ORCID http://orcid.org/0000-0002-5622-8069
Thiyagaraj Mayuranathan *Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.
Cicera R LazzarottoDepartment of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.ORCID http://orcid.org/0000-0003-1093-0175
Yichao LiDepartment of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.ORCID http://orcid.org/0000-0001-5791-096X
Heather Sheppard-TillmanDepartment of Pathology, St. Jude Children's Research Hospital, Memphis, TN, USA.ORCID http://orcid.org/0000-0002-9623-2812
Shaina N PorterDepartment of Cell and Molecular Biology, St. Jude Children's Research Hospital, Memphis, TN, USA.
Yu YaoDepartment of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.
Kalin MayberryDepartment of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.
Kelcee A EveretteMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA, USA.
Yoonjeong JangDepartment of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.
Christopher J PodrackyMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA, USA.
Elizabeth ThamanDivision of Hematology, Cancer and Blood Diseases Institute, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
Christophe LechauveDepartment of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.
Akshay SharmaDepartment of Bone Marrow Transplantation and Cellular Therapy, St Jude Children's Research Hospital, Memphis, TN, USA.ORCID http://orcid.org/0000-0003-3281-2081
Jordana M HendersonTriLink BioTechnologies, San Diego, CA, USA.
Michelle F RichterMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA, USA.
Kevin T ZhaoMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA, USA.ORCID http://orcid.org/0000-0003-3440-2094
Shannon M MillerMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA, USA.
Tina WangMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA, USA.
Luke W KoblanMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA, USA.ORCID http://orcid.org/0000-0003-2814-4412
Anton P McCaffreyTriLink BioTechnologies, San Diego, CA, USA.
John F TisdaleCellular and Molecular Therapeutics Branch, National Heart, Lung, and Blood Institute and National Institute of Diabetes and Digestive and Kidney Diseases, Bethesda, MD, USA.
Theodosia A KalfaDivision of Hematology, Cancer and Blood Diseases Institute, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.ORCID http://orcid.org/0000-0002-0426-9686
Shondra M Pruett-MillerDepartment of Cell and Molecular Biology, St. Jude Children's Research Hospital, Memphis, TN, USA.ORCID http://orcid.org/0000-0002-3793-585X
Shengdar Q TsaiDepartment of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.ORCID http://orcid.org/0000-0001-9161-3993
Mitchell J WeissDepartment of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA. mitch.weiss@stjude.org.ORCID http://orcid.org/0000-0003-2460-3036
David R LiuMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA, USA. drliu@fas.harvard.edu.ORCID http://orcid.org/0000-0002-9943-7557
St. Jude Children's Research Hospital · USBroad Institute · USCincinnati Children's Hospital Medical Center · USTriLink BioTechnologies (United States) · USNational Institute of Diabetes and Digestive and Kidney Diseases · US
Funding
VIROSOME/PROTEOLIPOSOME MEDIATED GENE TRANSFERP01HL053749 · NHLBI · ST. JUDE CHILDREN'S RESEARCH HOSPITAL · PI WEISS, MITCHELL J · 1994 to 2022
$43.6MCenter for Genomic Editing and Recording: Development and Application of Next-Generation Genome and Epigenome Editing Methods to Advance the Study and Treatment of Human DiseaseRM1HG009490 · NHGRI · WHITEHEAD INSTITUTE FOR BIOMEDICAL RES · PI Brittany S. Adamson, Martin Joseph Ankrah Aryee · 2017 to 2026
$22.7MIntegrating Chemistry and Evolution to Illuminate Biology and Enable Novel TherapeuticsR35GM118062 · NIGMS · HARVARD UNIVERSITY · PI LIU, DAVID R · 2016 to 2025
$6.4MPedGeneRx - Admin Supplement to Base Editing and Prime Editing for Sickle Cell Disease R01R01HL156647 · NHLBI · ST. JUDE CHILDREN'S RESEARCH HOSPITAL · PI Agnieszka Czechowicz, DAVID R LIU · 2021 to 2026
$4.8MContinuous Evolution of Proteins with Novel Therapeutic PotentialR01EB031172 · NIBIB · BROAD INSTITUTE, INC. · PI LIU, DAVID R · 2021 to 2024
$2.5MExpanding the Scope of Base EditingU01AI142756 · NIAID · BROAD INSTITUTE, INC. · PI LIU, DAVID R · 2018 to 2022
$2.1MA novel human T-cell platform to define biological effects of genome editingU01AI157189 · NIAID · ST. JUDE CHILDREN'S RESEARCH HOSPITAL · PI TSAI, SHENGDAR · 2020 to 2022
$1.8MContinuous Evolution of Proteins with Novel Therapeutic PotentialR01EB022376 · NIBIB · HARVARD UNIVERSITY · PI LIU, DAVID R · 2016 to 2019
$1.8MDoris Duke Charitable Foundation 2017093Howard Hughes Medical InstituteNHGRI NIH HHS RM1 HG009490NHLBI NIH HHS P01 HL053749NHLBI NIH HHS R01 HL156647NIAID NIH HHS U01 AI142756NIAID NIH HHS U01 AI157189NIBIB NIH HHS R01 EB022376NIBIB NIH HHS R01 EB031172NIGMS NIH HHS R35 GM118062
Abstract
Sickle cell disease (SCD) is caused by a mutation in the β-globin gene HBB
Indexed as
Gene EditingHematopoietic Stem Cell TransplantationAdenineAnemia, Sickle CellAnimalsAntigens, CD34beta-GlobinsCRISPR-Associated Protein 9Disease Models, AnimalFemaleGenetic TherapyGenome, HumanHematopoietic Stem CellsHumansMaleMiceAdenineAntigens, CD34beta-GlobinsCRISPR-Associated Protein 9
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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.