ArticleJournal of genetic syndromes & gene therapy2012
Liver-Directed Adeno-Associated Viral Gene Therapy for Hemophilia.
Article in Journal of genetic syndromes & gene therapy, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 22 papers.
What it found
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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.
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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.
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Who cites it
22 citing papers in PubMed, 31 citations in OpenAlex.
- Immune function in X-linked retinoschisis subjects in an AAV8-RS1 phase I/IIa gene therapy trial.Molecular therapy : the journal of the American Society of Gene Therapy · 2021Trial
- Viral vector-based gene therapies in the clinic: An update.Bioengineering & translational medicine · 2026Review
- Fat-1 Ameliorates Metabolic Dysfunction-Associated Fatty Liver Disease and Atherosclerosis through Promoting the Nuclear Localization of PPARα in Hamsters.Research (Washington, D.C.) · 2025Article
- Viral Vector Based Immunotherapy for Peanut Allergy.Viruses · 2024Review
- Miniaturization of hiPSC-derived 3D neural cultures in stirred-tank bioreactors for parallelized preclinical assessment of rAAV.Frontiers in bioengineering and biotechnology · 2024Article
- Next-generation strategies for gene-targeted therapies of central nervous system disorders: A workshop summary.Molecular therapy : the journal of the American Society of Gene Therapy · 2021Review
- CRISPR/Cas9 Mediated Deletion of the Angiotensinogen Gene Reduces Hypertension: A Potential for Cure?Hypertension (Dallas, Tex. : 1979) · 2021Article
- AAV-Mediated ApoC2 Gene Therapy: Reversal of Severe Hypertriglyceridemia and Rescue of Neonatal Death in ApoC2-Deficient Hamsters.Molecular therapy. Methods & clinical development · 2020Article
- Analysis of recombinant adeno-associated viral vector shedding in sheep following intracoronary delivery.Gene therapy · 2019Article
- Taste Receptor Cells in Mice Express Receptors for the Hormone Adiponectin.Chemical senses · 2019Article
- The adeno-associated virus rh10 vector is an effective gene transfer system for chronic spinal cord injury.Scientific reports · 2019Article
- Evaluation of engineered AAV capsids for hepatic factor IX gene transfer in murine and canine models.Journal of translational medicine · 2017Article
- Targeted approaches to induce immune tolerance for Pompe disease therapy.Molecular therapy. Methods & clinical development · 2016Review
- Potential for cellular stress response to hepatic factor VIII expression from AAV vector.Molecular therapy. Methods & clinical development · 2016Article
- Gene therapy for hemophilia.Frontiers in bioscience (Landmark edition) · 2015Review
- Scalable downstream strategies for purification of recombinant adeno- associated virus vectors in light of the properties.Current pharmaceutical biotechnology · 2015Review
- Copackaging of multiple adeno-associated viral vectors in a single production step.Human gene therapy methods · 2014Article
- Anti-CD20 as the B-Cell Targeting Agent in a Combined Therapy to Modulate Anti-Factor VIII Immune Responses in Hemophilia a Inhibitor Mice.Frontiers in immunology · 2014Article
- Portal vein delivery of viral vectors for gene therapy for hemophilia.Methods in molecular biology (Clifton, N.J.) · 2014Article
- Mechanistic insights into the enhancement of adeno-associated virus transduction by proteasome inhibitors.Journal of virology · 2013Article
Corrections and comments
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Authors and funding
2 authors at 1 institution in 1 country.
Funding
Abstract
Hemophilia A and B are monogenic bleeding disorders resulting from loss of functional coagulation factors VIII or IX, respectively. Prophylactic treatment requires frequent intravenous injections of exogenous factor VIII (F.VIII) or factor IX (F.IX), due to the short half-life of both factors. Hemophilia patients are at risk of developing neutralizing antibodies to F.VIII (~25-30%) or F.IX (~2-4%), which require the use of expensive bypass agents and immune tolerance induction protocols. Viral vector mediated liver gene transfer of F.VIII or F.IX offers an alternative treatment for hemophilia with easily defined clinical endpoints and no need for strict regulation of coagulation factor expression, as both proteins circulate as inactive zymogens. Adeno-associated viral (AAV) vectors are derived from a non-pathogenic human virus that efficiently transduce non-dividing cells, such as hepatocytes, and provide stable transgene expression.
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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.