ReviewResearch and practice in thrombosis and haemostasis2025
Gene therapy in hemophilia: the dawn of a new era.
Review in Research and practice in thrombosis and haemostasis, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 10 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
10 citing papers in PubMed.
- Roctavian withdrawal: exploring the gap between innovation and healthcare system readiness in hemophilia A gene therapy.Research and practice in thrombosis and haemostasis · 2026Review
- Screening chemical libraries for the development of oral treatments for bleeding disorders.Blood vessels, thrombosis & hemostasis · 2026Article
- Health care resources and costs associated with delivering gene therapy for hemophilia in clinical practice.Research and practice in thrombosis and haemostasis · 2026Article
- Nine areas with outstanding challenges for hemophilia B research.Therapeutic advances in hematology · 2026Review
- Quantification and comparison of anti-AAV9 and anti-AAVrh74 antibodies in plasma and human milk: Implications for AAV-based gene therapy candidacy.Journal of neuromuscular diseases · 2025Article
- How Close Are We to Achieving Durable and Efficacious Gene Therapy for Hemophilia A and B?Genes · 2025Review
- Curative Therapies for Hemophilias and Hemoglobinopathies in Adults: Immune, Gene, and Stem Cell Approaches in a Global Context.Biomedicines · 2025Review
- Altuvoct: Innovative Medicinal Products Benefit from Innovative Approaches to Regulatory Assessment.Life (Basel, Switzerland) · 2025Article
- Defining the Role of Nurses in Gene Therapy for Haemophilia.Haemophilia : the official journal of the World Federation of HemophiliaArticle
- Recent Advances in Gene Therapy for Hemophilia.Clinical and applied thrombosis/hemostasis : official journal of the International Academy of Clinical and Applied Thrombosis/HemostasisReview
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Hemophilia A and B are hereditary bleeding disorders associated with the X chromosome, stemming from genetic defects in the coding of coagulation factor (F)VIII or FIX protein, leading to partial or complete deficiency. In the absence of effective prophylaxis, these deficiencies can result in irreversible joint damage, known as hemophilic arthropathy, and subsequent disability. Despite advancements in hemophilia treatment, individuals with severe forms of the disease continue to face a high risk of bleeding, particularly in instances of trauma or major surgical procedures. In such scenarios, it remains imperative to administer replacement or bypassing drugs, especially when inhibitors are present. Within this context, gene therapy emerges as a compelling alternative, ensuring sustained expression of the deficient factor at levels often surpassing current recommendations. Some studies report an effect lasting up to 8 years, contributing significantly to clinical improvement and enhancing the quality of life for patients. However, a comprehensive evaluation of this innovative therapy is essential, encompassing both its benefits and potential risks. It is crucial to undertake a multidisciplinary assessment, engage in thoughtful discussions with the patient, and closely monitor the therapy's effects and any eventual side effects of therapy. This approach aims to facilitate an informed and collaborative decision-making process, ultimately maximizing the benefits for each individual patient.
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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.