Hum Gene Ther 2018 10;29(10):1183-1201
1 Aflac Cancer and Blood Disorders Center, Department of Pediatrics, School of Medicine, Emory University , Atlanta, Georgia; Christian Medical College , Vellore, India .
Genetically modified, autologous hematopoietic stem and progenitor cells (HSPCs) represent a new class of genetic medicine. Following this therapeutic paradigm, we are developing a product candidate, designated CD68-ET3-LV CD34, for the treatment of the severe bleeding disorder, hemophilia A. The product consists of autologous CD34 cells transduced with a human immunodeficiency virus 1-based, monocyte lineage-restricted, self-inactivating lentiviral vector (LV), termed CD68-ET3-LV, encoding a bioengineered coagulation factor VIII (fVIII) transgene, termed ET3, designed for enhanced expression. Read More