ArticleNucleic acids research2014
Nuclease-mediated gene editing by homologous recombination of the human globin locus.
Article in Nucleic acids research, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 62 papers.
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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
62 citing papers in PubMed, 95 citations in OpenAlex.
- The Molecular Pathology of Non-Malignant Haematological Disease.British journal of biomedical science · 2026Review
- MiR-223-3p regulates erythropoiesis by targeting TGFBR3/Smad signaling pathway in hemoglobin H-Constant Spring disease.Annals of medicine · 2025Article
- 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
- Ex vivo culture resting time impacts transplantation outcomes of genome-edited human hematopoietic stem and progenitor cells in xenograft mouse models.Cytotherapy · 2024Article
- Multiplex HDR for disease and correction modeling of SCID by CRISPR genome editing in human HSPCs.Molecular therapy. Nucleic acids · 2023Article
- Rare immune diseases paving the road for genome editing-based precision medicine.Frontiers in genome editing · 2023Review
- A Curative DNA Code for Hematopoietic Defects: Novel Cell Therapies for Monogenic Diseases of the Blood and Immune System.Hematology/oncology clinics of North America · 2022Review
- Gene Editing-Based Technologies forBiology · 2022Review
- Clinically relevant gene editing in hematopoietic stem cells for the treatment of pyruvate kinase deficiency.Molecular therapy. Methods & clinical development · 2021Article
- IPEX Syndrome: Genetics and Treatment Options.Genes · 2021Review
- Corrigendum: Targeted Gene Delivery: Where to Land.Frontiers in genome editing · 2021Article
- Gene Editing of Hematopoietic Stem Cells: Hopes and Hurdles Toward Clinical Translation.Frontiers in genome editing · 2021Review
- CRISPR-mediated gene modification of hematopoietic stem cells with beta-thalassemia IVS-1-110 mutation.Stem cell research & therapy · 2020Article
- A Preview of Selected Articles.Stem cells translational medicine · 2020Review
- Targeted Gene Delivery: Where to Land.Frontiers in genome editing · 2020Review
- Construction and Analysis of a Long Non-Coding RNA (lncRNA)-Associated ceRNA Network in β-Thalassemia and Hereditary Persistence of Fetal Hemoglobin.Medical science monitor : international medical journal of experimental and clinical research · 2019Article
- Editing the Sickle Cell Disease Mutation in Human Hematopoietic Stem Cells: Comparison of Endonucleases and Homologous Donor Templates.Molecular therapy : the journal of the American Society of Gene Therapy · 2019Article
- Gene correction for SCID-X1 in long-term hematopoietic stem cells.Nature communications · 2019Article
- Gene Therapy for Beta-Hemoglobinopathies: Milestones, New Therapies and Challenges.Molecular diagnosis & therapy · 2019Review
- Disruptive Technology: CRISPR/Cas-Based Tools and Approaches.Molecular diagnosis & therapy · 2019Review
2 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
4 authors at 2 institutions in 1 country.
Funding
Abstract
Tal-effector nucleases (TALENs) are engineered proteins that can stimulate precise genome editing through specific DNA double-strand breaks. Sickle cell disease and β-thalassemia are common genetic disorders caused by mutations in β-globin, and we engineered a pair of highly active TALENs that induce modification of 54% of human β-globin alleles near the site of the sickle mutation. These TALENS stimulate targeted integration of therapeutic, full-length beta-globin cDNA to the endogenous β-globin locus in 19% of cells prior to selection as quantified by single molecule real-time sequencing. We also developed highly active TALENs to human γ-globin, a pharmacologic target in sickle cell disease therapy. Using the β-globin and γ-globin TALENs, we generated cell lines that express GFP under the control of the endogenous β-globin promoter and tdTomato under the control of the endogenous γ-globin promoter. With these fluorescent reporter cell lines, we screened a library of small molecule compounds for their differential effect on the transcriptional activity of the endogenous β- and γ-globin genes and identified several that preferentially upregulate γ-globin expression.
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Registered trials
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.