ReviewBlood advances2017
Hemophilia gene therapy comes of age.
Review in Blood advances, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 38 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
38 citing papers in PubMed.
- Trial
- Therapeutic advances in hemophilia: from molecular innovation to patient-centered global care.Frontiers in medicine · 2025Review
- NF-κB-activated oncogene inhibition strategy for cancer gene therapy.Cancer gene therapy · 2024Article
- Optimizing liver health before and after gene therapy for hemophilia A.Blood advances · 2024Review
- Ultrasound-mediated gene delivery specifically targets liver sinusoidal endothelial cells for sustained FVIII expression in hemophilia A mice.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Article
- Binding and neutralizing anti-AAV antibodies: Detection and implications for rAAV-mediated gene therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2023Review
- A short hepatitis C virus NS5A peptide expression by AAV vector modulates human T cell activation and reduces vector immunogenicity.Gene therapy · 2022Article
- Transcutaneous ultrasound-mediated gene delivery into canine livers achieves therapeutic levels of factor VIII expression.Blood advances · 2022Article
- CRISPR-Cas9-Based Technology and Its Relevance to Gene Editing in Parkinson's Disease.Pharmaceutics · 2022Review
- Ultrasound-mediated gene delivery of factor VIII plasmids for hemophilia A gene therapy in mice.Molecular therapy. Nucleic acids · 2022Article
- International consensus recommendations on the management of people with haemophilia B.Therapeutic advances in hematology · 2022Review
- Advances in the management of haemophilia: emerging treatments and their mechanisms.Journal of biomedical science · 2021Review
- Optimizing language for effective communication of gene therapy concepts with hemophilia patients: a qualitative study.Orphanet journal of rare diseases · 2021Article
- The biphasic and age-dependent impact of klotho on hallmarks of aging and skeletal muscle function.eLife · 2021Article
- Platelet gene therapy induces robust immune tolerance even in a primed model via peripheral clonal deletion of antigen-specific T cells.Molecular therapy. Nucleic acids · 2021Article
- In vivo enrichment of genetically manipulated platelets for murine hemophilia B gene therapy.Journal of cellular physiology · 2021Article
- AAV vectors engineered to target insulin receptor greatly enhance intramuscular gene delivery.Molecular therapy. Methods & clinical development · 2020Article
- Enhancing therapeutic efficacy of in vivo platelet-targeted gene therapy in hemophilia A mice.Blood advances · 2020Article
- Defining the Optimal FVIII Transgene for Placental Cell-Based Gene Therapy to Treat Hemophilia A.Molecular therapy. Methods & clinical development · 2020Article
- Treatment of Hemophilia A Using Factor VIII Messenger RNA Lipid Nanoparticles.Molecular therapy. Nucleic acids · 2020Article
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.
Funding
Abstract
Concurrent with the development of recombinant factor replacement products, the characterization of the
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.