In Vivo HSC Gene Therapy Using a Bi-modular HDAd5/35++ Vector Cures Sickle Cell Disease in a Mouse Model
Chang Li, Hongjie Wang, Aphrodite Georgakopoulou, Sucheol Gil, Evangelia Yannaki, André Lieber
Topics & Concepts
In vivoGenetic enhancementVector (molecular biology)DiseaseBiologyModular designImmunologyViral vectorVectors in gene therapyVirologyMedicineGeneBiotechnologyGeneticsComputer sciencePathologyRecombinant DNAOperating systemVirus-based gene therapy researchCRISPR and Genetic EngineeringRNA Interference and Gene Delivery