ReviewTherapeutic advances in hematology2016
The potential of gene therapy approaches for the treatment of hemoglobinopathies: achievements and challenges.
Review in Therapeutic advances in hematology, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 20 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
20 citing papers in PubMed, 44 citations in OpenAlex.
- Strategies and innovations in hypertension management for sickle cell patients: A narrative review.Medicine · 2026Review
- Cellular and molecular targets in β-Thalassemia: advances in iron Regulation, Erythropoiesis, and Gene-Based therapies.Functional & integrative genomics · 2025Review
- Preclinical efficacy of a modified gamma-globin lentivirus gene therapy in Berkeley sickle cell anemia mice and human xenograft models.Molecular therapy. Methods & clinical development · 2025Article
- Combinatorial targeting of epigenome-modifying enzymes with decitabine and RN-1 synergistically increases HbF.Blood advances · 2023Article
- Non-Transfusion-Dependent Thalassemia: A Panoramic Review.Medicina (Kaunas, Lithuania) · 2022Review
- Joint efficacy of the three biomarkers SNCA, GYPB and HBG1 for atrial fibrillation and stroke: Analysis via the support vector machine neural network.Journal of cellular and molecular medicine · 2022Article
- Advancing the care of β-thalassaemia patients with novel therapies.Blood transfusion = Trasfusione del sangue · 2022Review
- Development and clinical translation ofComputational and structural biotechnology journal · 2022Review
- Genome-based therapeutic interventions for β-type hemoglobinopathies.Human genomics · 2021Review
- A 3D-bioprinted scaffold with doxycycline-controlled BMP2-expressing cells for inducing bone regeneration and inhibiting bacterial infection.Bioactive materials · 2021Article
- Ready for Repair? Gene Editing Enters the Clinic for the Treatment of Human Disease.Molecular therapy. Methods & clinical development · 2020Review
- CRISPR-mediated gene modification of hematopoietic stem cells with beta-thalassemia IVS-1-110 mutation.Stem cell research & therapy · 2020Article
- TALEN-Mediated Gene Editing ofMolecular therapy. Methods & clinical development · 2019Article
- Potential role of LSD1 inhibitors in the treatment of sickle cell disease: a review of preclinical animal model data.American journal of physiology. Regulatory, integrative and comparative physiology · 2018Review
- Sickle Cell Anemia and Its Phenotypes.Annual review of genomics and human genetics · 2018Review
- Molecular basis of β thalassemia and potential therapeutic targets.Blood cells, molecules & diseases · 2018Review
- Simple chronic transfusion therapy, a crucial therapeutic option for sickle cell disease, improves but does not normalize blood rheology: What should be our goals for transfusion therapy?Clinical hemorheology and microcirculation · 2018Article
- A Universal Approach to Correct Various HBB Gene Mutations in Human Stem Cells for Gene Therapy of Beta-Thalassemia and Sickle Cell Disease.Stem cells translational medicine · 2018Article
- Article
- Diagnostic approaches for inherited hemolytic anemia in the genetic era.Blood research · 2017Review
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
Abstract
Hemoglobinopathies, including β-thalassemia and sickle cell disease (SCD), are a heterogeneous group of commonly inherited disorders affecting the function or levels of hemoglobin. Disease phenotype can be severe with substantial morbidity and mortality. Bone marrow transplantation is curative, but limited to those patients with an appropriately matched donor. Genetic therapy, which utilizes a patient's own cells, is thus an attractive therapeutic option. Numerous therapies are currently in clinical trials or in development, including therapies utilizing gene replacement therapy using lentiviruses and the latest gene editing techniques. In addition, methods are being developed that may be able to expand gene therapies to those with poor access to medical care, potentially significantly decreasing the global burden of 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.