ArticleCell reports. Medicine2021
Preclinical evaluation for engraftment of CD34
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Article in Cell reports. Medicine, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 24 papers.
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24citing papers in PubMed
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Who cites it
24 citing papers in PubMed, 27 citations in OpenAlex.
- Engraftment Outcome of CRISPR/Cas9-Edited Hematopoietic Stem Cells for Genetic Diseases: A Systematic Review and Meta-Analysis of Preclinical Evidence.Journal of hematology · 2026Article
- Development of evolutionarily conserved viral integration sites as safe harbors for human gene therapy.iScience · 2025Article
- Gene Therapy of Beta Hemoglobinopathies.Biomedicines · 2025Review
- Hematopoietic stem cell therapy with gene modification to treat sickle cell disease.Stem cells translational medicine · 2025Review
- Current and future treatments for sickle cell disease: From hematopoietic stem cell transplantation to in vivo gene therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
- Genome editing strategies for targeted correction of β-globin mutation in sickle cell disease: From bench to bedside.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
- A bioinformatic analysis of gene editing off-target loci altered by common polymorphisms, using 'PopOff'.Journal of the Royal Society of New Zealand · 2025Article
- CRISPR technology in human diseases.MedComm · 2024Review
- Challenges and innovations in hematopoietic stem cell transplantation: exploring bone marrow niches and new model systems.BMB reports · 2024Review
- A simultaneous knockout knockin genome editing strategy in HSPCs potently inhibits CCR5- and CXCR4-tropic HIV-1 infection.Cell stem cell · 2024Article
- Impact of CRISPR/HDR editing versus lentiviral transduction on long-term engraftment and clonal dynamics of HSPCs in rhesus macaques.Cell stem cell · 2024Article
- Development of pathophysiologically relevant models of sickle cell disease and β-thalassemia for therapeutic studies.Nature communications · 2024Article
- Precise genome-editing in human diseases: mechanisms, strategies and applications.Signal transduction and targeted therapy · 2024Review
- Fertility-preserving myeloablative conditioning using single-dose CD117 antibody-drug conjugate in a rhesus gene therapy model.Nature communications · 2023Article
- Defining global strategies to improve outcomes in sickle cell disease: a Lancet Haematology Commission.The Lancet. Haematology · 2023Review
- Pre-existing immunity does not impair the engraftment of CRISPR-Cas9-edited cells in rhesus macaques conditioned with busulfan or radiation.Molecular therapy. Methods & clinical development · 2023Article
- Forced enhancer-promoter rewiring to alter gene expression in animal models.Molecular therapy. Nucleic acids · 2023Article
- A culture platform to study quiescent hematopoietic stem cells following genome editing.Cell reports methods · 2022Article
- Review
- Nonhuman Primates in Translational Research.Annual review of animal biosciences · 2022Review
Corrections and comments
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Authors and funding
18 authors at 3 institutions in 2 countries.
Naoya UchidaCellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institutes (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD, USA.
Linhong LiMaxCyte, Gaithersburg, MD, USA.
Tina NassehiCellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institutes (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD, USA.
Claire M DrysdaleCellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institutes (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD, USA.
Morgan YapundichCellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institutes (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD, USA.
Jackson GamerCellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institutes (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD, USA.
Juan J Haro-MoraCellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institutes (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD, USA.
Selami DemirciCellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institutes (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD, USA.
Alexis LeonardCellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institutes (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD, USA.
Aylin C BonifacinoTranslational Stem Cell Biology Branch, NHLBI, NIH, Bethesda, MD, USA.
Allen E KrouseTranslational Stem Cell Biology Branch, NHLBI, NIH, Bethesda, MD, USA.
N Seth LindeTranslational Stem Cell Biology Branch, NHLBI, NIH, Bethesda, MD, USA.
Cornell AllenMaxCyte, Gaithersburg, MD, USA.
Madhusudan V PeshwaMaxCyte, Gaithersburg, MD, USA.
Suk See De RavinLaboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases (NIAID), NIH, Bethesda, MD, USA.
Robert E DonahueCellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institutes (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD, USA.
Harry L MalechLaboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases (NIAID), NIH, Bethesda, MD, USA.
John F TisdaleCellular and Molecular Therapeutics Branch, National Heart Lung and Blood Institutes (NHLBI)/National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Institutes of Health (NIH), Bethesda, MD, USA.
National Institutes of Health · USMaxCyte (United States) · USNational Institute of Allergy and Infectious Diseases · US
Funding
Retroviral Mediated Gene Transfer Into Primate Hematopoietic CellsZIAHL002338 · NHLBI · NATIONAL HEART, LUNG, AND BLOOD INSTITUTE · PI HONG, SO GUN · 2019 to 2025
$26.3MGene Therapy Basic Research to Treat Inherited Primary Immune DeficienciesZIAAI000644 · NIAID · NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES · PI MALECH, HARRY L · 2009 to 2025
$12.1MDevelopment and Clinical Application of Gene and Cell therapies for Patients with Immune DeficienciesZIAAI000645 · NIAID · NATIONAL INSTITUTE OF ALLERGY AND INFECTIOUS DISEASES · PI DE RAVIN, SUK SEE · 2009 to 2025
$10.1MAbstract
Sickle cell disease (SCD) is caused by a 20A > T mutation in the β-globin gene. Genome-editing technologies have the potential to correct the SCD mutation in hematopoietic stem cells (HSCs), producing adult hemoglobin while simultaneously eliminating sickle hemoglobin. Here, we developed high-efficiency viral vector-free non-footprint gene correction in SCD CD34
Indexed as
Anemia, Sickle CellAnimalsAntigens, CD34beta-GlobinsGene EditingGene TargetingHematopoietic Stem CellsHematopoietic Stem Cell TransplantationHemoglobin, SickleHeterograftsHumansMacaca mulattaMiceRNA, Guide, CRISPR-Cas SystemsAntigens, CD34beta-GlobinsHemoglobin, SickleRNA, Guide, CRISPR-Cas SystemsCRISPR/Cas9electroporationgene correctiongenome editinghematopoietic stem celllarge animal modelnon-human primatesickle cell diseasetransplantationβ-globin gene
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LicenceCC BY-NC-ND
Read underepoch 390
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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.