ArticleNature medicine2019
Highly efficient therapeutic gene editing of human hematopoietic stem cells.
Abstract read
In one paragraph
Article in Nature medicine, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 316 papers, 2 of them syntheses that pooled it.
0numbers the graph read from it
0cells of the map it votes in
316citing papers in PubMed, 2 pooled it
25.4field-weighted citation impact, top 1% of its field
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
316 citing papers in PubMed, 2 syntheses or guidelines pooled it, 509 citations in OpenAlex.
- Evidence Synthesis of Gene Therapy and Gene Editing from Different Disorders-Implications for Individuals with Rett Syndrome: A Systematic Review.International journal of molecular sciences · 2023Pooled it
- Using Clustered Regularly Interspaced Short Palindromic Repeats gene editing to induce permanent expression of fetal hemoglobin in β-thalassemia and sickle cell disease: A comparative meta-analysis.Frontiers in medicine · 2022Pooled it
- Zinc finger nuclease-mediated gene editing in hematopoietic stem cells results in reactivation of fetal hemoglobin in sickle cell disease.Scientific reports · 2024Trial
- RNA therapeutics: current status and future directions.Signal transduction and targeted therapy · 2026Review
- Culturing neutrophils from human CD34STAR protocols · 2026Article
- Current Status of Cellular and Gene-Based Therapies for Congenital Metabolic Disorders: A Review.Medical science monitor : international medical journal of experimental and clinical research · 2026Review
- Co-delivery of lentiviral vectors and Cas9-containing virus-like particles enables rapid, scalable manufacture of gene-edited CAR T cells.Molecular therapy. Advances · 2026Article
- Nuclear remodeling and optimal migration emerge early, and constriction-passage progressively improves during hematopoietic stem cells to neutrophils differentiation.bioRxiv : the preprint server for biology · 2026Article
- Engineered lipid nanoparticles for in vivo and durable editing of haematopoietic stem cells within humanized mice.Nature biomedical engineering · 2026Article
- Incorporating AI-optimized zinc finger proteins enhances the efficiencies and targeting ranges of miniature base editors.Nature communications · 2026Article
- SORT LNPs encapsulating Cas9 mRNA achieve efficient editing in skeletal muscle in a dystrophic mouse model.Molecular therapy : the journal of the American Society of Gene Therapy · 2026Article
- Deep learning-guided engineering of SpuFz1 and rational miniaturization of ωRNA enables efficient genome editing.Nature communications · 2026Article
- Selection of human hematopoietic stem cells bearing the intended functional edit by transient AND-gate reporters.Nature biotechnology · 2026Article
- Fully Modified SpyCas9 Guide RNAs Enable Robust Genome Editing In Cells and In Vivo.bioRxiv : the preprint server for biology · 2026Article
- Establishment of an immortalized erythroid model for Hb Bart's hydrops fetalis with homozygous αScientific reports · 2026Article
- Engineered dCas12f1-SAM enables robust transcriptional activation and gain-of-function screening in primary human cells.Nature communications · 2026Article
- Article
- Clinical Trial Landscape of Gene-Edited Autologous Hematopoietic Stem Cells for Hemoglobinopathies and Immunodeficiencies.International journal of molecular sciences · 2026Review
- Calcium Shock Enables Efficient and Programmable Particle Delivery for Genome Editing Applications.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2026Article
- Article
256 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
24 authors at 5 institutions in 3 countries.
Yuxuan WuDivision of Hematology/Oncology, Boston Children's Hospital , Boston, MA, USA.
Jing ZengDivision of Hematology/Oncology, Boston Children's Hospital , Boston, MA, USA.
Benjamin P RoscoeDepartment of Molecular, Cell and Cancer Biology, Li Weibo Institute for Rare Diseases Research, University of Massachusetts Medical School, Worcester, MA, USA.
Pengpeng LiuDepartment of Molecular, Cell and Cancer Biology, Li Weibo Institute for Rare Diseases Research, University of Massachusetts Medical School, Worcester, MA, USA.
Qiuming YaoDivision of Hematology/Oncology, Boston Children's Hospital , Boston, MA, USA.
Cicera R LazzarottoDepartment of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.
Kendell ClementMolecular Pathology Unit, Center for Cancer Research, and Center for Computational and Integrative Biology, Massachusetts General Hospital, Boston, MA, USA.
Mitchel A ColeDivision of Hematology/Oncology, Boston Children's Hospital , Boston, MA, USA.
Kevin LukDepartment of Molecular, Cell and Cancer Biology, Li Weibo Institute for Rare Diseases Research, University of Massachusetts Medical School, Worcester, MA, USA.
Cristina BaricordiDivision of Hematology/Oncology, Boston Children's Hospital , Boston, MA, USA.
Anne H ShenDivision of Hematology/Oncology, Boston Children's Hospital , Boston, MA, USA.
Chunyan RenDivision of Hematology/Oncology, Boston Children's Hospital , Boston, MA, USA.
Erica B EsrickDivision of Hematology/Oncology, Boston Children's Hospital , Boston, MA, USA.
John P ManisDivision of Hematology/Oncology, Boston Children's Hospital , Boston, MA, USA.
David M DorfmanDepartment of Pathology, Brigham and Women's Hospital , Boston, MA, USA.
David A WilliamsDivision of Hematology/Oncology, Boston Children's Hospital , Boston, MA, USA.
Alessandra BiffiDivision of Hematology/Oncology, Boston Children's Hospital , Boston, MA, USA.
Carlo BrugnaraDivision of Hematology/Oncology, Boston Children's Hospital , Boston, MA, USA.ORCID http://orcid.org/0000-0001-8192-8713
Luca BiascoDivision of Hematology/Oncology, Boston Children's Hospital , Boston, MA, USA.
Christian BrendelDivision of Hematology/Oncology, Boston Children's Hospital , Boston, MA, USA.
Luca PinelloMolecular Pathology Unit, Center for Cancer Research, and Center for Computational and Integrative Biology, Massachusetts General Hospital, Boston, MA, USA.
Shengdar Q TsaiDepartment of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.
Scot A WolfeDepartment of Molecular, Cell and Cancer Biology, Li Weibo Institute for Rare Diseases Research, University of Massachusetts Medical School, Worcester, MA, USA.
Daniel E BauerDivision of Hematology/Oncology, Boston Children's Hospital , Boston, MA, USA. daniel.bauer@childrens.harvard.edu.ORCID http://orcid.org/0000-0001-5076-7945
Broad Institute · USUniversity of Massachusetts Chan Medical School · USSt. Jude Children's Research Hospital · USBoston Children's Hospital · USBrigham and Women's Hospital · US
Funding
Harvard Clinical and Translational Science CenterUL1TR001102 · NCATS · HARVARD MEDICAL SCHOOL · PI NADLER, LEE MARSHALL · 2013 to 2017
$106.2MVIROSOME/PROTEOLIPOSOME MEDIATED GENE TRANSFERP01HL053749 · NHLBI · ST. JUDE CHILDREN'S RESEARCH HOSPITAL · PI RYU, BYOUNG Y · 1994 to 2022
$43.6MTRANSCRIPTION FACTOR GATA-1 IN TERMINAL ERYTHROID DEVELOPMENTP01HL032262 · NHLBI · CHILDREN'S HOSPITAL BOSTON · PI ORKIN, STUART H · 1985 to 2021
$38.9MStem Cell and Transplantation BiologyU54DK106829 · NIDDK · FRED HUTCHINSON CANCER RESEARCH CENTER · PI DEREK L STIREWALT · 2015 to 2026
$9.1MTranslational and clinical studies targeting y-globin modulationU01HL117720 · NHLBI · BOSTON CHILDREN'S HOSPITAL · PI SANKARAN, VIJAY GANESH · 2013 to 2017
$7.4MGene therapy targeting BCL11A to induce fetal hemoglobin and reduce sickle hemoglobin in patients with Sickle Cell DiseaseR01HL137848 · NHLBI · BOSTON CHILDREN'S HOSPITAL · PI WILLIAMS, DAVID A · 2017 to 2021
$6.0MNext generation hybrid nucleases for precise excision of latent HIV-1 provirusR01AI117839 · NIAID · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI LUBAN, JEREMY, WOLFE, SCOT A · 2015 to 2019
$4.6MHigh-throughput discovery of essential noncoding sequences for erythropoiesisDP2HL137300 · NHLBI · BOSTON CHILDREN'S HOSPITAL · PI BAUER, DANIEL EVAN · 2016 to 2016
$2.7MEngineered Cas9 Nucleases with Single-Genomic-Site Precision for CYBB CorrectionR01GM115911 · NIGMS · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI SONTHEIMER, ERIK J., WOLFE, SCOT A · 2016 to 2019
$1.6MDiscovering disease-causal variants by linking genetic and epigenetic variationR00HG008399 · NHGRI · MASSACHUSETTS GENERAL HOSPITAL · PI PINELLO, LUCA · 2017 to 2019
$747kEpigenetic regulation of BCL11A in the hemoglobin switchK08DK093705 · NIDDK · BOSTON CHILDREN'S HOSPITAL · PI BAUER, DANIEL EVAN · 2012 to 2016
$679kAcoustic separation of peripheral blood for cellular therapy of sickle cell anemiaR21HL145636 · NHLBI · BOSTON CHILDREN'S HOSPITAL · PI MANIS, JOHN P · 2019 to 2020
$541kDoris Duke Charitable Foundation 2013137Doris Duke Charitable Foundation 2015134NCATS NIH HHS UL1 TR001102NHGRI NIH HHS R00 HG008399NHLBI NIH HHS DP2 HL137300NHLBI NIH HHS P01 HL032262NHLBI NIH HHS P01 HL053749NHLBI NIH HHS R01 HL137848NHLBI NIH HHS R21 HL145636NHLBI NIH HHS U01 HL117720NIAID NIH HHS R01 AI117839NIDDK NIH HHS K08 DK093705NIDDK NIH HHS R03 DK109232NIDDK NIH HHS U54 DK106829NIGMS NIH HHS R01 GM115911
Abstract
Re-expression of the paralogous γ-globin genes (HBG1/2) could be a universal strategy to ameliorate the severe β-globin disorders sickle cell disease (SCD) and β-thalassemia by induction of fetal hemoglobin (HbF, α
Indexed as
Amino Acid SequenceAnemia, Sickle CellBase Sequencebeta-ThalassemiaCarrier ProteinsCRISPR-Cas SystemsEnhancer Elements, GeneticErythroid Precursor CellsFetal Hemoglobingamma-GlobinsGene EditingHematopoietic Stem CellsHematopoietic Stem Cell TransplantationHumansINDEL MutationNuclear ProteinsBCL11A protein, humanCarrier ProteinsFetal Hemoglobingamma-GlobinsNuclear ProteinsRepressor ProteinsRNA, Guide, CRISPR-Cas Systems
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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.