ArticleNature biomedical engineering2023
Ex vivo prime editing of patient haematopoietic stem cells rescues sickle-cell disease phenotypes after engraftment in mice.
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Article in Nature biomedical engineering, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 99 papers.
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0cells of the map it votes in
99citing papers in PubMed
19.5field-weighted citation impact, top 1% of its field
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Who cites it
99 citing papers in PubMed, 127 citations in OpenAlex.
- Prime Editing: An Overview.Methods in molecular biology (Clifton, N.J.) · 2027Review
- MN•CS•IO System: Cross-Species Study for HBB Function in High-Altitude Hypoxia Adaptation.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2026Article
- Culturing neutrophils from human CD34STAR protocols · 2026Article
- Article
- Beyond Precision: A Multidimensional Framework for Selecting Genetic Medicine Platforms.Cells · 2026Review
- Dense RNA motif modifications enable robust in vivo prime editing and enhance efficiencies of diverse editing systems.Nature biomedical engineering · 2026Article
- Enhanced γ-globin reactivation and sickle cell correction through a repressor-to-activator motif switch in theMolecular therapy. Nucleic acids · 2026Article
- Gene editing of hematopoietic stem cells: applications and advances.International journal of hematology · 2026Review
- Article
- Advances in prime editing: Molecular innovations, Large-fragment engineering, and AI-driven design.Biodesign research · 2026Review
- Compound delivery of eVLPs enhances prime editing for targeted genome engineering and high-throughput screening.Cell genomics · 2026Article
- Mechanistic machine learning for prediction of prime editing outcomes.Nature biotechnology · 2026Article
- CRISPR application in hematological disorders: from bench to bedside.Blood advances · 2026Review
- Engineered lipid nanoparticles for in vivo and durable editing of haematopoietic stem cells within humanized mice.Nature biomedical engineering · 2026Article
- Therapeutic Gene Editing of APOE4 in Sporadic Alzheimer's Disease via Prime Editor 7.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2026Article
- Delivery Systems for Therapeutic Genome Editing: Challenges, Innovations, and Future Perspectives.MedComm · 2026Review
- Advancing Hematopoietic Stem Cell Research: The Impact of Bioengineered 3D Bone Marrow Niches.Stem cell reviews and reports · 2026Review
- Efficient prime editing in vivo and in vitro using lipid nanoparticles.Nature nanotechnology · 2026Article
- Review
- A primer on prime: A prime editing update from advances to first-in-human trial.Molecular therapy : the journal of the American Society of Gene Therapy · 2026Review
39 more citing papers are in PubMed but not listed here.
Corrections and comments
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Authors and funding
20 authors at 3 institutions in 1 country.
Kelcee A EveretteMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA, USA.ORCID http://orcid.org/0000-0003-4421-971X
Gregory A NewbyMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA, USA.ORCID http://orcid.org/0000-0001-7869-2615
Rachel M LevineDepartment of Hematology, St Jude Children's Research Hospital, Memphis, TN, USA.ORCID http://orcid.org/0000-0002-3684-2950
Kalin MayberryDepartment of Hematology, St Jude Children's Research Hospital, Memphis, TN, USA.
Yoonjeong JangDepartment of Hematology, St Jude Children's Research Hospital, Memphis, TN, USA.
Thiyagaraj MayuranathanDepartment of Hematology, St Jude Children's Research Hospital, Memphis, TN, USA.ORCID http://orcid.org/0000-0002-9505-0363
Nikitha NimmagaddaDepartment of Hematology, St Jude Children's Research Hospital, Memphis, TN, USA.ORCID http://orcid.org/0000-0003-1648-6335
Erin DempseyDepartment of Hematology, St Jude Children's Research Hospital, Memphis, TN, USA.
Yichao LiDepartment of Hematology, St Jude Children's Research Hospital, Memphis, TN, USA.
Senthil Velan BhoopalanDepartment of Hematology, St Jude Children's Research Hospital, Memphis, TN, USA.ORCID http://orcid.org/0000-0002-1402-6549
Xiong LiuMolecular and Clinical Hematology Branch, National Heart, Lung, and Blood Institute/National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health, Bethesda, MD, USA.
Jessie R DavisMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA, USA.
Andrew T NelsonMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA, USA.
Peter J ChenMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA, USA.
Alexander A SousaMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA, USA.
Yong ChengDepartment of Hematology, St Jude Children's Research Hospital, Memphis, TN, USA.ORCID http://orcid.org/0000-0002-9314-0971
John F TisdaleMolecular and Clinical Hematology Branch, National Heart, Lung, and Blood Institute/National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health, Bethesda, MD, USA.
Mitchell J WeissDepartment of Hematology, St Jude Children's Research Hospital, Memphis, TN, USA.ORCID http://orcid.org/0000-0002-6734-4858
Jonathan S YenDepartment of Hematology, St Jude Children's Research Hospital, Memphis, TN, USA. jonathan.yen@stjude.org.ORCID http://orcid.org/0000-0002-9432-9450
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 · USNational Institutes of Health · US
Funding
Viral Vector Technology (VVTSR)P30CA021765 · NCI · ST. JUDE CHILDREN'S RESEARCH HOSPITAL · PI Shondra Michelle Miller · 1985 to 2026
$166.9MVIROSOME/PROTEOLIPOSOME MEDIATED GENE TRANSFERP01HL053749 · NHLBI · ST. JUDE CHILDREN'S RESEARCH HOSPITAL · PI RYU, BYOUNG Y · 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
$22.7MNovel Gene Editing Approaches for HemoglobinopathiesR01HL136135 · NHLBI · FRED HUTCHINSON CANCER RESEARCH CENTER · PI HANS-PETER KIEM · 2017 to 2026
$7.7MIntegrating 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
$4.8MExpanding the Scope of Base EditingU01AI142756 · NIAID · BROAD INSTITUTE, INC. · PI LIU, DAVID R · 2018 to 2022
$2.1MHoward Hughes Medical InstituteNCI NIH HHS P30 CA021765NHGRI NIH HHS RM1 HG009490NHLBI NIH HHS P01 HL053749NHLBI NIH HHS R01 HL136135NHLBI NIH HHS R01 HL156647NIAID NIH HHS U01 AI142756NIGMS NIH HHS R35 GM118062
Abstract
Sickle-cell disease (SCD) is caused by an A·T-to-T·A transversion mutation in the β-globin gene (HBB). Here we show that prime editing can correct the SCD allele (HBB
Indexed as
Anemia, Sickle CellGene EditingAdultAnimalsbeta-GlobinsCRISPR-Cas SystemsDNAHematopoietic Stem CellsHumansMicePhenotypebeta-GlobinsDNA
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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 Socratic graph.