Peer-reviewed veterinary case report
Gene therapy stops most bleeding in dogs with severe hemophilia
By Callan, Mary Beth et al.·Published in PloS one·2016·Department of Clinical Studies, United States·View original on PubMed →
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Original publication title: Successful Phenotype Improvement following Gene Therapy for Severe Hemophilia A in Privately Owned Dogs.
- Species:
- dog
Plain-English summary
Two privately owned dogs with severe hemophilia A, a bleeding disorder, were treated with gene therapy that involved delivering a modified virus to their liver. This therapy successfully increased their clotting factor levels to about 1-2% of normal, which significantly reduced their bleeding episodes by 90%. One of the dogs had a specific genetic mutation linked to the condition. Overall, the treatment showed promising results, improving the dogs' health and quality of life.
People also search for: dog hemophilia A treatment · gene therapy for dogs · bleeding disorder in dogs · dog liver gene therapy
Abstract
Severe hemophilia A (HA) is an inherited bleeding disorder characterized by <1% of residual factor VIII (FVIII) clotting activity. The disease affects several mammals including dogs, and, like humans, is associated with high morbidity and mortality. In gene therapy using adeno-associated viral (AAV) vectors, the canine model has been one of the best predictors of the therapeutic dose tested in clinical trials for hemophilia B (factor IX deficiency) and other genetic diseases, such as congenital blindness. Here we report our experience with liver gene therapy with AAV-FVIII in two outbred, privately owned dogs with severe HA that resulted in sustained expression of 1-2% of normal FVIII levels and prevented 90% of expected bleeding episodes. A Thr62Met mutation in the F8 gene was identified in one dog. These data recapitulate the improvement of the disease phenotype in research animals, and in humans, with AAV liver gene therapy for hemophilia B. Our experience is a novel example of the benefits of a relevant preclinical canine model to facilitate both translational studies in humans and improved welfare of privately owned dogs.
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Search related cases →Original publication on PubMed: https://pubmed.ncbi.nlm.nih.gov/27011017/