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

por Carbonaro-Sarracino, Denise A., Chun, Krista, Clark, Danielle N., Kaufman, Michael L., Jin, Xiangyang, Wang, Xiaoyan, Kohn, Donald B.
Publicado 2021

In Situ Spatial Reconstruction of Distinct Normal and Pathological Cell Populations Within the Human Adrenal Gland

por Fu, Rui, Walters, Kathryn, Kaufman, Michael L, Koc, Katrina, Baldwin, Amber, Clay, Michael R, Basham, Kaitlin J, Kiseljak-Vassiliades, Katja, Fishbein, Lauren, Mukherjee, Neelanjan
Publicado 2023

The human ankyrin 1 promoter insulator sustains gene expression in a β-globin lentiviral vector in hematopoietic stem cells

por Romero, Zulema, Campo-Fernandez, Beatriz, Wherley, Jennifer, Kaufman, Michael L, Urbinati, Fabrizia, Cooper, Aaron R, Hoban, Megan D, Baldwin, Kismet M, Lumaquin, Dianne, Wang, Xiaoyan, Senadheera, Shantha, Hollis, Roger P, Kohn, Donald B
Publicado 2015

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

por Carbonaro-Sarracino, Denise A., Tarantal, Alice F., Lee, C. Chang I., Kaufman, Michael L., Wandro, Stephen, Jin, Xiangyang, Martinez, Michele, Clark, Danielle N., Chun, Krista, Koziol, Colin, Hardee, Cinnamon L., Wang, Xiaoyan, Kohn, Donald B.
Publicado 2019