ArticleHuman gene therapy2016
Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the β(A(T87Q))-Globin Gene.
Article in Human gene therapy, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 79 papers.
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.
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Who cites it
79 citing papers in PubMed, 153 citations in OpenAlex.
- Sotatercept, a novel transforming growth factor β ligand trap, improves anemia in β-thalassemia: a phase II, open-label, dose-finding study.Haematologica · 2019Trial
- The landscape and trajectory of global CRISPR therapeutics.Molecular therapy. Nucleic acids · 2026Review
- A review of genetic modification for ex vivo cellular therapies.Transfusion · 2026Article
- Emerging Considerations in Transfusion Medicine: Hematopoietic Stem and Progenitor Cell Collection for Gene Therapy for Sickle Cell Disease and Transfusion-Dependent Thalassemia.Clinics in laboratory medicine · 2026Review
- Advances in Gene Therapy for Inherited Haemoglobinopathies.Hematology reports · 2025Review
- An erythroid-specific lentiviral vector improves anemia and iron metabolism in a new model of XLSA.Blood · 2025Article
- Emerging Gene Therapies in Sickle Cell Disease: A Comparative Review of Efficacy and Safety Against Standard Treatments.Journal of blood medicine · 2025Review
- Revolutionary breakthrough: FDA approves CASGEVY, the first CRISPR/Cas9 gene therapy for sickle cell disease.Annals of medicine and surgery (2012) · 2024Review
- Regulatory Assessment of Casgevy for the Treatment of Transfusion-Dependent β-Thalassemia and Sickle Cell Disease with Recurrent Vaso-Occlusive Crises.Current issues in molecular biology · 2024Review
- Autologous gene therapy for hemoglobinopathies: From bench to patient's bedside.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Review
- Defining global strategies to improve outcomes in sickle cell disease: a Lancet Haematology Commission.The Lancet. Haematology · 2023Review
- Clinical applications of gene therapy for rare diseases: A review.International journal of experimental pathology · 2023Review
- Gene Therapy and Gene Editing for β-Thalassemia.Hematology/oncology clinics of North America · 2023Review
- Nanotechnology-enabled gene delivery for cancer and other genetic diseases.Expert opinion on drug delivery · 2023Review
- Review
- Research and Therapeutic Approaches in Stem Cell Genome Editing by CRISPR Toolkit.Molecules (Basel, Switzerland) · 2023Review
- Lovo-cel gene therapy for sickle cell disease: Treatment process evolution and outcomes in the initial groups of the HGB-206 study.American journal of hematology · 2023Article
- RS12574989 and haplotype associated with α/β-chain imbalance and population HbA2 reduction.BMC medical genomics · 2022Article
- Development and clinical translation ofComputational and structural biotechnology journal · 2022Review
- Recent advances in lentiviral vectors for gene therapy.Science China. Life sciences · 2021Review
19 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
11 authors at 5 institutions in 3 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
β-globin gene disorders are the most prevalent inherited diseases worldwide and result from abnormal β-globin synthesis or structure. Novel therapeutic approaches are being developed in an effort to move beyond palliative management. Gene therapy, by ex vivo lentiviral transfer of a therapeutic β-globin gene derivative (β(AT87Q)-globin) to hematopoietic stem cells, driven by cis-regulatory elements that confer high, erythroid-specific expression, has been evaluated in human clinical trials over the past 8 years. β(AT87Q)-globin is used both as a strong inhibitor of HbS polymerization and as a biomarker. While long-term studies are underway in multiple centers in Europe and in the United States, proof-of-principle of efficacy and safety has already been obtained in multiple patients with β-thalassemia and sickle cell disease.
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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.