Sustained Correction of OTC Deficiency in Spf (ash) mice Using Optimized Self-complementary AAV2/8 Vectors

por Wang, Lili, Wang, Huan, Morizono, Hiroki, Bell, Peter, Jones, David, Lin, Jianping, McMenamin, Deirdre, Yu, Hongwei, Batshaw, Mark L., Wilson, James M.
Publicado 2011

Determining the Minimally Effective Dose of a Clinical Candidate AAV Vector in a Mouse Model of Crigler-Najjar Syndrome

por Greig, Jenny A., Nordin, Jayme M.L., Draper, Christine, McMenamin, Deirdre, Chroscinski, Edward A., Bell, Peter, Gray, John T., Richman, Laura K., Wilson, James M.
Publicado 2018

A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice

por Yang, Yang, Wang, Lili, Bell, Peter, McMenamin, Deirdre, He, Zhenning, White, John, Yu, Hongwei, Xu, Chenyu, Morizono, Hiroki, Musunuru, Kiran, Batshaw, Mark L., Wilson, James M.
Publicado 2016

A mutation-independent CRISPR-Cas9–mediated gene targeting approach to treat a murine model of ornithine transcarbamylase deficiency

por Wang, Lili, Yang, Yang, Breton, Camilo, Bell, Peter, Li, Mingyao, Zhang, Jia, Che, Yan, Saveliev, Alexei, He, Zhenning, White, John, Latshaw, Caitlin, Xu, Chenyu, McMenamin, Deirdre, Yu, Hongwei, Morizono, Hiroki, Batshaw, Mark L., Wilson, James M.
Publicado 2020

Adeno-associated virus-mediated expression of human butyrylcholinesterase to treat organophosphate poisoning

por Gupta, Vibhor, Cadieux, C. Linn, McMenamin, Deirdre, Medina-Jaszek, C. Angelica, Arif, Muhammad, Ahonkhai, Omua, Wielechowski, Erik, Taheri, Maryam, Che, Yan, Goode, Tamara, Limberis, Maria P., Li, Mingyao, Cerasoli, Douglas M., Tretiakova, Anna P., Wilson, James M.
Publicado 2019

DWI Metrics Differentiating Benign Intraductal Papillary Mucinous Neoplasms from Invasive Pancreatic Cancer: A Study in GEM Models

por Romanello Joaquim, Miguel, Furth, Emma E., Fan, Yong, Song, Hee Kwon, Pickup, Stephen, Cao, Jianbo, Choi, Hoon, Gupta, Mamta, Cao, Quy, Shinohara, Russell, McMenamin, Deirdre, Clendenin, Cynthia, Karasic, Thomas B., Duda, Jeffrey, Gee, James C., O’Dwyer, Peter J., Rosen, Mark A., Zhou, Rong
Publicado 2022