ReviewHematology. American Society of Hematology. Education Program2021
Hemophilia gene therapy: ushering in a new treatment paradigm?
Review in Hematology. American Society of Hematology. Education Program, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 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
14 citing papers in PubMed, 1 synthesis or guideline pooled it, 23 citations in OpenAlex.
- Liver-related aspects of valoctocogene roxaparvovec gene therapy for hemophilia A: expert guidance for clinical practice.Blood advances · 2024Guideline
- Non-viral gene therapy for hemophilia A: long-term outcomes of minicircle FVIII delivery in a mouse model.Frontiers in pharmacology · 2026Article
- Chinese guidance for the clinical application of adeno-associated virus vector-based gene therapy for hemophilia B (2025).Blood science (Baltimore, Md.) · 2025Article
- Preclinical Evaluation of AAV8-R338L Gene Therapy for Hemophilia: Efficacy, Pharmacokinetics, Distribution, Excretion and Toxicity in Mouse Models and Non-human Primates.Pharmaceutical research · 2025Article
- Gene therapy as an innovative approach to the treatment of hemophilia B-a review.Journal of applied genetics · 2025Review
- [Chinese guidance for the clinical application of Adeno-associated virus vector-based gene therapy for hemophilia B (2025)].Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi · 2025Article
- Consensus-Based Expert Recommendations on the Management of Hemophilia A in the Gulf Region.Acta haematologica · 2025Review
- Gene and cell therapy of human genetic diseases: Recent advances and future directions.Journal of cellular and molecular medicine · 2024Review
- Correlation of antigen expression with epigenetic modifications after rAAV delivery of a human factor IX variant in mice and rhesus macaques.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Article
- Efficient gene transduction in pigs and macaques with the engineered AAV vector AAV.GT5 for hemophilia B gene therapy.Molecular therapy. Methods & clinical development · 2023Article
- Adeno-Associated Virus Gene Therapy for Hemophilia.Annual review of medicine · 2023Review
- Haemophilia gene therapy: experiences and lessons from treated patients.Orphanet journal of rare diseases · 2022Article
- Adeno-associated virus-mediated expression of activated factor V (FVa) for hemophilia phenotypic correction.Frontiers in medicine · 2022Article
- Preoperative diagnosis and management of inherited bleeding disorders in female adolescents and adults.Canadian journal of surgery. Journal canadien de chirurgieReview
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
1 author at 1 institution in 1 country.
Funding
Abstract
After 3 decades of clinical trials, repeated proof-of-concept success has now been demonstrated in hemophilia A and B gene therapy. Current clinical hemophilia gene therapy efforts are largely focused on the use of systemically administered recombinant adeno-associated viral (rAAV) vectors for F8 or F9 gene addition. With multiple ongoing trials, including licensing studies in hemophilia A and B, many are cautiously optimistic that the first AAV vectors will obtain regulatory approval within approximately 1 year. While supported optimism suggests that the goal of gene therapy to alter the paradigm of hemophilia care may soon be realized, a number of outstanding questions have emerged from clinical trial that are in need of answers to harness the full potential of gene therapy for hemophilia patients. This article reviews the use of AAV vector gene addition approaches for hemophilia A and B, focusing specifically on information to review in the process of obtaining informed consent for hemophilia patients prior to clinical trial enrollment or administering a licensed AAV vector.
Indexed as
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.