Safe and efficient in vivo hematopoietic stem cell transduction in nonhuman primates using HDAd5/35++ vectors
Chang Li, Hongjie Wang, Sucheol Gil, Audrey Germond, Connie Fountain, Audrey Baldessari, Jiho Kim, Zhinan Liu, Aphrodite Georgakopoulou, Stefan Radtke, Tamás Raskó, Amit Pande, Christina Chiang, Eli Chin, Evangelia Yannaki, Zsuzsanna Izsvák, Thalia Papayannopoulou, Hans‐Peter Kiem, André Lieber
Abstract
HSC gene therapy could be feasible in humans without the need for high-dose chemotherapy conditioning and HSC transplantation.
Topics & Concepts
BiologyHaematopoiesisStem cellIn vivoSpleenImmunologyGenetic enhancementTransplantationHematopoietic stem cellBone marrowViral vectorTransgeneMolecular biologyCell biologyMedicineInternal medicineGeneticsGeneRecombinant DNAVirus-based gene therapy researchRNA Interference and Gene DeliveryParvovirus B19 Infection Studies