ReviewInternational journal of molecular sciences2021
Gene Therapy in Hemophilia: Recent Advances.
Review in International journal of molecular sciences, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 24 papers, 1 of them a synthesis that pooled it.
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
24 citing papers in PubMed, 1 synthesis or guideline pooled it, 52 citations in OpenAlex.
- Global trends in hemophilic arthropathy research: a bibliometric and visualization analysis.Frontiers in medicine · 2025Pooled it
- Gene Therapy in Hemophilia: Clinical Advances, Immunological Challenges, and Emerging Therapeutic Perspectives.International journal of molecular sciences · 2026Review
- Treatment of canine hemophilia A via intraosseous delivery of a platelet-specific factor VIII-lentiviral vector.Blood vessels, thrombosis & hemostasis · 2026Article
- Advancements in CRISPR-basedFrontiers in genome editing · 2026Review
- How Close Are We to Achieving Durable and Efficacious Gene Therapy for Hemophilia A and B?Genes · 2025Review
- Clinical perspective: Advancing hemophilia treatment through gene therapy approaches.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
- Lymphoblastoid andFrontiers in bioengineering and biotechnology · 2025Article
- Impact of Replacement Therapy on Pregnancy Outcomes in Hemophilia Carriers: A Historical Cohort Study in Saudi Arabia.Life (Basel, Switzerland) · 2024Article
- Delivery of DNA-Based Therapeutics for Treatment of Chronic Diseases.Pharmaceutics · 2024Review
- C and G are frequently mutated into T and A in coding regions of human genes.Molecular genetics and genomics : MGG · 2024Article
- Forecasting the potential impact of cell and gene therapies in France: projecting product launches and patients treated.Frontiers in medicine · 2024Article
- Liver injury in cynomolgus monkeys following intravenous and intrathecal scAAV9 gene therapy delivery.Molecular therapy : the journal of the American Society of Gene Therapy · 2023Article
- Molecular pathogenesis of a novel Met394Thr variant causing hemophilia B.Molecular genetics & genomic medicine · 2023Article
- Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research priorities to transform the care of people with hemophilia.Expert review of hematology · 2023Article
- From a bispecific monoclonal antibody to gene therapy: A new era in the treatment of hemophilia A.Biomedical papers of the Medical Faculty of the University Palacky, Olomouc, Czechoslovakia · 2023Review
- Cerebellar hematoma in severe hemophilia with inhibitor on emicizumab prophylaxis: a case report.Journal of medical case reports · 2023Article
- Correction ofFrontiers in genetics · 2023Article
- The Arrival of Gene Therapy for Patients with Hemophilia A.International journal of molecular sciences · 2022Review
- The Vascular Endothelium and Coagulation: Homeostasis, Disease, and Treatment, with a Focus on the Von Willebrand Factor and Factors VIII and V.International journal of molecular sciences · 2022Review
- Rare Monogenic Diseases: Molecular Pathophysiology and Novel Therapies.International journal of molecular sciences · 2022Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Hemophilia is a monogenic mutational disease affecting coagulation factor VIII or factor IX genes. The palliative treatment of choice is based on the use of safe and effective recombinant clotting factors. Advanced therapies will be curative, ensuring stable and durable concentrations of the defective circulating factor. Results have so far been encouraging in terms of levels and times of expression using mainly adeno-associated vectors. However, these therapies are associated with immunogenicity and hepatotoxicity. Optimizing the vector serotypes and the transgene (variants) will boost clotting efficacy, thus increasing the viability of these protocols. It is essential that both physicians and patients be informed about the potential benefits and risks of the new therapies, and a register of gene therapy patients be kept with information of the efficacy and long-term adverse events associated with the treatments administered. In the context of hemophilia, gene therapy may result in (particularly indirect) cost savings and in a more equitable allocation of treatments. In the case of hemophilia A, further research is needed into how to effectively package the large factor VIII gene into the vector; and in the case of hemophilia B, the priority should be to optimize both the vector serotype, reducing its immunogenicity and hepatotoxicity, and the transgene, boosting its clotting efficacy so as to minimize the amount of vector administered and decrease the incidence of adverse events without compromising the efficacy of the protein expressed.
Indexed as
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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.