Gene delivery using AAV8 in vivo for disease stabilization in a bimodal gene therapy approach for the treatment of ADA-deficient SCID

by Denise A. Carbonaro-Sarracino, Krista Chun, Danielle N. Clark, Michael L. Kaufman, Xiangyang Jin, Xiaoyan Wang, Donald B. Kohn
Published 2021-03-01

Dosing and Re-Administration of Lentiviral Vector for In Vivo Gene Therapy in Rhesus Monkeys and ADA-Deficient Mice

by Denise A. Carbonaro-Sarracino, Alice F. Tarantal, C. Chang I. Lee, Michael L. Kaufman, Stephen Wandro, Xiangyang Jin, Michele Martinez, Danielle N. Clark, Krista Chun, Colin Koziol, Cinnamon L. Hardee, Xiaoyan Wang, Donald B. Kohn
Published 2020-03-01