Peer-reviewed veterinary case report
Complete correction of hemophilia B phenotype by FIX-Padua skeletal muscle gene therapy in an inhibitor-prone dog model.
- Journal:
- Blood advances
- Year:
- 2018
- Authors:
- French, Robert A et al.
- Affiliation:
- Department of Pediatrics
- Species:
- dog
Abstract
Skeletal muscle–directed expression of FIX-Padua resulted in complete correction of HB phenotype in an inhibitor-prone dog model. Long-term immune tolerance to FIX is sustained over years upon multiple challenges with recombinant FIX protein in 2 HB models.
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Search related cases →Original publication: https://pubmed.ncbi.nlm.nih.gov/29500218/