Abstract Book of Joint 10th BIC and 3rd INHIBITORS in Hemophilia International Conference Genoa, Italy, September 6–8 2019

摘要

Introduction: Liver-directed gene therapy with adeno-associated viral (AAV) vectors delivering a clotting factor transgene into hepatocytes has shown successful results in adults with hemophilia. However, be- cause AAV vectors do not actively integrate into the host cell genome, they are diluted upon cell division during liver growth, thus challeng- ing their proficient use in pediatric patients. In contrast, lentiviral vec- tors (LV) integrate into the target cell chromatin and are maintained as cells divide.