ReviewHuman gene therapy. Clinical development2015
Translational data from adeno-associated virus-mediated gene therapy of hemophilia B in dogs.
Review in Human gene therapy. Clinical development, 2015. 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.
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
22 citing papers in PubMed, 35 citations in OpenAlex.
- Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant.The New England journal of medicine · 2017Trial
- Comparing molecular and computational approaches for detecting viral integration of AAV gene therapy constructs.Molecular therapy. Methods & clinical development · 2023Article
- Adeno-associated virus infection and its impact in human health: an overview.Virology journal · 2022Review
- Evaluating the state of the science for adeno-associated virus integration: An integrated perspective.Molecular therapy : the journal of the American Society of Gene Therapy · 2022Review
- Evolutionary insights into coagulation factor IX Padua and other high-specific-activity variants.Blood advances · 2021Article
- CRISPR/Cas9-mediated knockin of human factor IX into swine factor IX locus effectively alleviates bleeding in hemophilia B pigs.Haematologica · 2021Article
- Complexity of immune responses to AAV transgene products - Example of factor IX.Cellular immunology · 2019Review
- AAV2/9-mediated overexpression of MIF inhibits SOD1 misfolding, delays disease onset, and extends survival in mouse models of ALS.Proceedings of the National Academy of Sciences of the United States of America · 2019Article
- Bridging from Intramuscular to Limb Perfusion Delivery of rAAV: Optimization in a Non-human Primate Study.Molecular therapy. Methods & clinical development · 2019Article
- Gene Therapy With Regulatory T Cells: A Beneficial Alliance.Frontiers in immunology · 2018Review
- Hemophilia gene therapy comes of age.Blood advances · 2017Review
- Hemophilia gene therapy comes of age.Hematology. American Society of Hematology. Education Program · 2017Review
- Gene Therapy for Hemophilia.Molecular therapy : the journal of the American Society of Gene Therapy · 2017Review
- Evaluation of engineered AAV capsids for hepatic factor IX gene transfer in murine and canine models.Journal of translational medicine · 2017Article
- Systemic AAV Gene Therapy Close to Clinical Trials for Several Neuromuscular Diseases.Molecular therapy : the journal of the American Society of Gene Therapy · 2017Article
- Article
- Genetic Manipulation of Brown Fat Via Oral Administration of an Engineered Recombinant Adeno-associated Viral Serotype Vector.Molecular therapy : the journal of the American Society of Gene Therapy · 2016Article
- Sustained correction of FVII deficiency in dogs using AAV-mediated expression of zymogen FVII.Blood · 2016Article
- Successful Phenotype Improvement following Gene Therapy for Severe Hemophilia A in Privately Owned Dogs.PloS one · 2016Article
- Adeno-Associated Virus Type 2 and Hepatocellular Carcinoma?Human gene therapy · 2015Article
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 5 institutions in 1 country.
Funding
Abstract
Preclinical testing of new therapeutic strategies in relevant animal models is an essential part of drug development. The choice of animal models of disease that are used in these studies is driven by the strength of the translational data for informing about safety, efficacy, and success or failure of human clinical trials. Hemophilia B is a monogenic, X-linked, inherited bleeding disorder that results from absent or dysfunctional coagulation factor IX (FIX). Regarding preclinical studies of adeno-associated virus (AAV)-mediated gene therapy for hemophilia B, dogs with severe hemophilia B (<1% FIX) provide well-characterized phenotypes and genotypes in which a species-specific transgene can be expressed in a mixed genetic background. Correction of the hemophilic coagulopathy by sustained expression of FIX, reduction of bleeding events, and a comprehensive assessment of the humoral and cell-mediated immune responses to the expressed transgene and recombinant AAV vector are all feasible end points in these dogs. This review compares the preclinical studies of AAV vectors used to treat dogs with hemophilia B with the results obtained in subsequent human clinical trials using muscle- and liver-based approaches.
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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.