Muscle-Directed Delivery of an AAV1 Vector Leads to Capsid-Specific T Cell Exhaustion in Nonhuman Primates and Humans

por Gernoux, Gwladys, Gruntman, Alisha M., Blackwood, Meghan, Zieger, Marina, Flotte, Terence R., Mueller, Christian
Publicado 2020

Liver-directed SERPINA1 gene therapy attenuates progression of spontaneous and tobacco smoke-induced emphysema in α1-antitrypsin null mice

por Zieger, Marina, Borel, Florie, Greer, Cynthia, Gernoux, Gwladys, Blackwood, Meghan, Flotte, Terence R., Mueller, Christian
Publicado 2022

Modulating immune responses to AAV by expanded polyclonal T-regs and capsid specific chimeric antigen receptor T-regulatory cells

por Arjomandnejad, Motahareh, Sylvia, Katelyn, Blackwood, Meghan, Nixon, Thomas, Tang, Qiushi, Muhuri, Manish, Gruntman, Alisha M., Gao, Guangping, Flotte, Terence R., Keeler, Allison M.
Publicado 2021

CRISPR/Cas9-mediated excision of ALS/FTD-causing hexanucleotide repeat expansion in C9ORF72 rescues major disease mechanisms in vivo and in vitro

por Meijboom, Katharina E., Abdallah, Abbas, Fordham, Nicholas P., Nagase, Hiroko, Rodriguez, Tomás, Kraus, Carolyn, Gendron, Tania F., Krishnan, Gopinath, Esanov, Rustam, Andrade, Nadja S., Rybin, Matthew J., Ramic, Melina, Stephens, Zachary D., Edraki, Alireza, Blackwood, Meghan T., Kahriman, Aydan, Henninger, Nils, Kocher, Jean-Pierre A., Benatar, Michael, Brodsky, Michael H., Petrucelli, Leonard, Gao, Fen-Biao, Sontheimer, Erik J., Brown, Robert H., Zeier, Zane, Mueller, Christian
Publicado 2022