ArticleNature genetics2023
Potent and uniform fetal hemoglobin induction via base editing.
Open access · greenAbstract read
In one paragraph
Article in Nature genetics, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 54 papers.
0numbers the graph read from it
0cells of the map it votes in
54citing papers in PubMed
19.8field-weighted citation impact, top 1% of its field
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
54 citing papers in PubMed, 69 citations in OpenAlex.
- Lentiviral gene therapy with reduced-intensity conditioning for sickle cell disease: a phase 1/2 trial.Nature medicine · 2025Trial
- Therapeutic upregulation of gene expression in inherited cardiomyopathies from current approaches to future directions.Nature cardiovascular research · 2026Review
- Base editing for precision therapeutics.Cell genomics · 2026Review
- 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
- Mechanistic machine learning for prediction of prime editing outcomes.Nature biotechnology · 2026Article
- Article
- Genetic influences on haematopoiesis.Nature reviews. Genetics · 2026Review
- Review
- Therapeutic adenine base editor with minimized off-target effects.Protein & cell · 2026Article
- Article
- Combinatorial base editing couples disease correction with lineage amplification in hematopoietic stem and progenitor cells.bioRxiv : the preprint server for biology · 2026Article
- Mechanistic machine learning enables interpretable and generalizable prediction of prime editing outcomes.bioRxiv : the preprint server for biology · 2026Article
- Microfluidic capillary transit velocity as a functional measure for sickle cell disease andLab on a chip · 2026Article
- Review
- Sensitive and unbiased genome-wide profiling of base-editor-induced off-target activity using CHANGE-seq-BE.Nature biotechnology · 2026Article
- Advances in Gene Therapy for Inherited Haemoglobinopathies.Hematology reports · 2025Review
- Gene Therapy of Beta Hemoglobinopathies.Biomedicines · 2025Review
- Lipid nanoparticle mediated base editing of the Q344X rhodopsin mutation associated with retinitis pigmentosa.Gene therapy · 2025Article
- G2B: an optimized lentiviral vector with enhanced titer and β-globin expression for improved β-thalassemia gene therapy.Blood science (Baltimore, Md.) · 2025Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
27 authors at 4 institutions in 2 countries.
Thiyagaraj Mayuranathan *Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.ORCID 0000-0002-9505-0363
Gregory A Newby *Merkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA, USA.ORCID 0000-0001-7869-2615
Ruopeng FengDepartment of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.
Yu YaoDepartment of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.
Kalin D MayberryDepartment 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 0000-0003-1093-0175
Yichao LiDepartment of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.ORCID 0000-0001-5791-096X
Rachel M LevineDepartment of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.ORCID 0000-0002-3684-2950
Nikitha NimmagaddaDepartment of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.ORCID 0000-0003-1648-6335
Erin DempseyDepartment of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.ORCID 0009-0000-2512-0939
Guolian KangDepartment of Biostatistics, St. Jude Children's Research Hospital, Memphis, TN, USA.ORCID 0000-0002-6102-0115
Shaina N PorterDepartment of Cell and Molecular Biology, St. Jude Children's Research Hospital, Memphis, TN, USA.
Phillip A DoerflerDepartment of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.ORCID 0000-0001-7566-9073
Jingjing ZhangDepartment of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.
Yoonjeong JangDepartment of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.
Jingjing ChenDepartment of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.ORCID 0000-0003-4179-0371
Henry W BellSchool of Biotechnology and Biomolecular Sciences, University of New South Wales, Sydney, New South Wales, Australia.ORCID 0000-0003-4677-8208
Merlin CrossleySchool of Biotechnology and Biomolecular Sciences, University of New South Wales, Sydney, New South Wales, Australia.ORCID 0000-0003-2057-3642
Senthil Velan BhoopalanDepartment of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.ORCID 0000-0002-1402-6549
Akshay SharmaDepartment of Bone Marrow Transplantation and Cellular Therapy, St. Jude Children's Research Hospital, Memphis, TN, USA.ORCID 0000-0003-3281-2081
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.
Shondra M Pruett-MillerDepartment of Cell and Molecular Biology, St. Jude Children's Research Hospital, Memphis, TN, USA.ORCID 0000-0002-3793-585X
Yong ChengDepartment of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.ORCID 0000-0002-9314-0971
Shengdar Q TsaiDepartment of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA.ORCID 0000-0001-9161-3993
David R LiuMerkin Institute of Transformative Technologies in Healthcare, Broad Institute of Harvard and MIT, Cambridge, MA, USA. drliu@fas.harvard.edu.ORCID 0000-0002-9943-7557
Mitchell J WeissDepartment of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA. mitch.weiss@stjude.org.ORCID 0000-0003-2460-3036
Jonathan S YenDepartment of Hematology, St. Jude Children's Research Hospital, Memphis, TN, USA. jonathan.yen@stjude.org.ORCID 0000-0002-9432-9450
St. Jude Children's Research Hospital · USBroad Institute · USUNSW Sydney · AUNational Institute of Diabetes and Digestive and Kidney Diseases · 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
$4.8MIdentification of causal non-coding variants in childhood genetic disordersR35GM133614 · NIGMS · ST. JUDE CHILDREN'S RESEARCH HOSPITAL · PI CHENG, YONG · 2019 to 2023
$2.2MExpanding 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
$1.8MDefining the mechanisms of hemoglobin switching and genotoxicities associated with its manipulationK01DK132453 · NIDDK · VERSITI BLOOD HEALTH, INC. · PI Phillip A Doerfler · 2022 to 2026
$760kIn vivo precision genome editing to correct genetic diseaseR00HL163805 · NHLBI · JOHNS HOPKINS UNIVERSITY · PI NEWBY, GREGORY A. · 2023 to 2025
$747kHoward Hughes Medical InstituteNCI NIH HHS P30 CA021765NHGRI NIH HHS RM1 HG009490NHLBI NIH HHS K99 HL163805NHLBI NIH HHS P01 HL053749NHLBI NIH HHS R00 HL163805NHLBI NIH HHS R01 HL136135NHLBI NIH HHS R01 HL156647NIAID NIH HHS U01 AI142756NIAID NIH HHS U01 AI157189NIBIB NIH HHS R01 EB022376NIDDK NIH HHS K01 DK132453NIGMS NIH HHS R35 GM118062NIGMS NIH HHS R35 GM133614
Abstract
Inducing fetal hemoglobin (HbF) in red blood cells can alleviate β-thalassemia and sickle cell disease. We compared five strategies in CD34
Indexed as
Anemia, Sickle Cellbeta-ThalassemiaAnimalsAntigens, CD34Fetal Hemoglobingamma-GlobinsGene EditingMiceAntigens, CD34Fetal Hemoglobingamma-Globins
Identifiers
What OpenQuestion holds
Textmetadata
LicenceCC BY
Read underepoch 390
Registered trials
None linked
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.