liver directed lentiviral gene therapy in a dog model of hemophilia b

• DOI: 10.17615/gbc7-vx32
• Publication Date: 2015-03-04
• Authors: Manfred Schmidt; Monica Volpin; Alessio Cantore; Francesca Sanvito; Patrizia Della Valle; Dwight A. Bellinger; Francesca Bellintani; Robin A. Raymer; Fabrizio Benedicenti; Lucia Sergi Sergi; Armando D'Angelo; Elizabeth P. Merr...; Claudio Doglioni; Marco Ranzani; Thierry VandenDri...; Luigi Naldini; Pierangela Gallina; Eugenio Montini; Marinee Chuah; Marinee Chuah; Timothy C. Nichols; Cynthia C. Bartho...; Samia Martin
• Keywords: 0301 basic medicine; Time Factors; [SDV]Life Sciences [q-bio]; Genetic Vectors; Lentivirus; Gene Therapy; Genetic Therapy; Sciences bio-médicales et agricoles; Hemophilia B; 3. Good health; [SDV] Life Sciences [q-bio]; Mice, Inbred C57BL; Disease Models, Animal; 03 medical and health sciences; Dogs; Liver; Transduction, Genetic; Animals; Female; Transgenes; Blood Coagulation; Mutagens

Gene therapy with lentiviral vectors targeting transgene expression to hepatocytes provides stable reconstitution of clotting activity in dogs with hemophilia B and does not show genotoxicity in tumor-prone mice.