Sustained correction of disease in naive and AAV2-pretreated hemophilia B dogs: AAV2/8-mediated, liver-directed gene therapy.
Wang L, Calcedo R, Nichols TC, Bellinger DA, Dillow A, Verma IM, Wilson JM
Efficient transduction of liver and muscle after in utero injection of lentiviral vectors with different pseudotypes.
MacKenzie TC, Kobinger GP, Kootstra NA, Radu A, Sena-Esteves M, Bouchard S, Wilson JM, Verma IM, Flake AW
Molecular Therapy : The Journal Of The American Society Of Gene Therapy