Optimized Adeno-Associated Viral-Mediated Human Factor VIII Gene Therapy in Cynomolgus Macaques
| Autor: | Tamara Goode, James M. Wilson, Jenny A. Greig, Roberto Calcedo, Samuel C. Wadsworth, Erin Bote, John W White, Jayme M.L. Nordin, Lili Wang, Qiang Wang |
|---|---|
| Rok vydání: | 2018 |
| Předmět: |
0301 basic medicine
business.industry viruses Genetic enhancement medicine.disease_cause Virology 03 medical and health sciences 030104 developmental biology 0302 clinical medicine Text mining hemic and lymphatic diseases 030220 oncology & carcinogenesis Genetics medicine Molecular Medicine business Human Blood Coagulation Factor Molecular Biology Adeno-associated virus |
| Zdroj: | Human gene therapy. |
| ISSN: | 1557-7422 |
| Popis: | Hemophilia A is a common hereditary bleeding disorder that is characterized by a deficiency of human blood coagulation factor VIII (hFVIII). Previous studies with adeno-associated viral (AAV) vectors identified two liver-specific promoter and enhancer combinations (E03.TTR and E12.A1AT) that drove high level expression of a codon-optimized, B-domain-deleted hFVIII transgene in a mouse model of the disease. This study further evaluated these enhancer/promoter combinations in cynomolgus macaques using two different AAV capsids (AAVrh10 and AAVhu37). Each of the four vector combinations was administered intravenously at a dose of 1.2 × 10 |
| Databáze: | OpenAIRE |
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