ArticleJournal of genetic syndromes & gene therapy2011
Engineering Factor Viii for Hemophilia Gene Therapy.
Article in Journal of genetic syndromes & gene therapy, 2011. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 14 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
14 citing papers in PubMed, 15 citations in OpenAlex.
- Deconstructing gene therapy in hemophilia for the clinician.Hematology. American Society of Hematology. Education Program · 2025Review
- Advances in biopharmaceutical products for hemophilia.iScience · 2024Review
- A Critical Exploration of the Total Flavonoid Content Assay for Honey.Methods and protocols · 2024Article
- Evaluating Gene Therapy as a Potential Paradigm Shift in Treating Severe Hemophilia.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2023Review
- Ultrasound-mediated gene delivery of factor VIII plasmids for hemophilia A gene therapy in mice.Molecular therapy. Nucleic acids · 2022Article
- Development of an AAV DNA-based synthetic vector for the potential gene therapy of hemophilia in children.Frontiers in microbiology · 2022Article
- Minimal Essential Human Factor VIII Alterations Enhance Secretion and Gene Therapy Efficiency.Molecular therapy. Methods & clinical development · 2020Article
- Identification of Key Coagulation Activity Determining Elements in Canine Factor VIII.Molecular therapy. Methods & clinical development · 2020Article
- Plant cell-made protein antigens for induction of Oral tolerance.Biotechnology advances · 2019Review
- CRISPR therapeutic tools for complex genetic disorders and cancer (Review).International journal of oncology · 2018Review
- Cornerstones of CRISPR-Cas in drug discovery and therapy.Nature reviews. Drug discovery · 2017Review
- mRNA-based therapeutics--developing a new class of drugs.Nature reviews. Drug discovery · 2014Review
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Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
6 authors at 1 institution in 1 country.
Funding
Abstract
Current treatment of hemophilia A by intravenous infusion of factor VIII (fVIII) concentrates is very costly and has a potential adverse effect of developing inhibitors. Gene therapy, on the other hand, can potentially overcome these limitations associated with fVIII replacement therapy. Although hemophilia B gene therapy has achieved promising outcomes in human clinical trials, hemophilia A gene therapy lags far behind. Compared to factor IX, fVIII is a large protein which is difficult to express at sustaining therapeutic levels when delivered by either viral or non-viral vectors. To improve fVIII gene delivery, numerous strategies have been exploited to engineer the fVIII molecule and overcome the hurdles preventing long term and high level expression. Here we reviewed these strategies, and discussed their pros and cons in human gene therapy of hemophilia A.
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.