ReviewHemaSphere2020
Gene Therapy of the Hemoglobinopathies.
Review in HemaSphere, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
14 citing papers in PubMed, 33 citations in OpenAlex.
- Epidemiology and economic burden of selected rare genetic diseases in Germany - a claims database study.Orphanet journal of rare diseases · 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
- Clinical Complications and Healthcare Resource Utilization Associated with Conventional Management of Sickle Cell Disease with Recurrent Vaso-occlusive Crises and Transfusion-Dependent β-Thalassemia in Germany.PharmacoEconomics - open · 2025Article
- Sickle cell disease: understanding pathophysiology, clinical features and advances in gene therapy approaches.Frontiers in pharmacology · 2025Review
- Sickle Cell Disease.Transfusion medicine and hemotherapy : offizielles Organ der Deutschen Gesellschaft fur Transfusionsmedizin und Immunhamatologie · 2024Review
- Viral and Non-Viral Systems to Deliver Gene Therapeutics to Clinical Targets.International journal of molecular sciences · 2024Review
- Understanding Rare Anemias: Emerging Frontiers for Diagnosis and Treatment.Journal of clinical medicine · 2024Review
- Novel frontiers in gene therapy: In vivo gene editing.HemaSphere · 2024Article
- Gene Editing-Based Technologies forBiology · 2022Review
- Does the world need germline editing for β-thalassemia?Haematologica · 2022Article
- The EHA Research Roadmap: Hematopoietic Stem Cell Gene Therapy.HemaSphere · 2022Article
- HSCT remains the only cure for patients with transfusion-dependent thalassemia until gene therapy strategies are proven to be safe.Bone marrow transplantation · 2021Review
- Genome-based therapeutic interventions for β-type hemoglobinopathies.Human genomics · 2021Review
- Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Sickle cell disease and the ß-thalassemias are caused by mutations of the ß-globin gene and represent the most frequent single gene disorders worldwide. Even in European countries with a previous low frequency of these conditions the prevalence has substantially increased following large scale migration from Africa and the Middle East to Europe. The hemoglobin diseases severely limit both, life expectancy and quality of life and require either life-long supportive therapy if cure cannot be achieved by allogeneic stem cell transplantation. Strategies for
Identifiers
What OpenQuestion holds
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.