Overexpression of factor VIII after AAV delivery is transiently associated with cellular stress in hemophilia A mice

por Lange, Amy M, Altynova, Ekaterina S, Nguyen, Giang N, Sabatino, Denise E
Publicado 2016

Evaluating the state of the science for adeno-associated virus integration: An integrated perspective

por Sabatino, Denise E., Bushman, Frederic D., Chandler, Randy J., Crystal, Ronald G., Davidson, Beverly L., Dolmetsch, Ricardo, Eggan, Kevin C., Gao, Guangping, Gil-Farina, Irene, Kay, Mark A., McCarty, Douglas M., Montini, Eugenio, Ndu, Adora, Yuan, Jing
Publicado 2022

A long-term study of AAV gene therapy in hemophilia A dogs identifies clonal expansions of transduced liver cells

por Nguyen, Giang N., Everett, John K., Kafle, Samita, Roche, Aoife M., Raymond, Hayley E., Leiby, Jacob, Wood, Christian, Assenmacher, Charles-Antoine, Merricks, Elizabeth P., Long, C. Tyler, Kazazian, Haig H., Nichols, Timothy C., Bushman, Frederic D., Sabatino, Denise E.
Publicado 2020

Preclinical assessment of an optimized AAV-FVIII vector in mice and non-human primates for the treatment of hemophilia A

por Elkouby, Liron, Armour, Sean M., Toso, Raffaella, DiPietro, Marti, Davidson, Robert J., Nguyen, Giang N., Willet, Mallory, Kutza, Stephanie, Silverberg, Joseph, Frick, Jennifer, Crosariol, Marco, Wang, Yuhuan, Wang, Chuansong, High, Katherine A., Sabatino, Denise E., Anguela, Xavier M.
Publicado 2021

Functionalized lipid-like nanoparticles for in vivo mRNA delivery and base editing

por Zhang, Xinfu, Zhao, Weiyu, Nguyen, Giang N., Zhang, Chengxiang, Zeng, Chunxi, Yan, Jingyue, Du, Shi, Hou, Xucheng, Li, Wenqing, Jiang, Justin, Deng, Binbin, McComb, David W., Dorkin, Robert, Shah, Aalok, Barrera, Luis, Gregoire, Francine, Singh, Manmohan, Chen, Delai, Sabatino, Denise E., Dong, Yizhou
Publicado 2020