The Rare Disease Research Scholars Program: A training curriculum for clinical researchers with mixed methods evaluation study

por Regier, Debra S., Weaver, Jennifer A., Cheng, Nancy, Batshaw, Mark L., Ottolini, Mary, Shy, Michael E., Summar, Marshall L.
Publicado 2022

The remarkable journey of one female individual with ornithine transcarbamylase deficiency diagnosed post‐mortem

por Forsyth, RaeLynn, Peretz, Ryan H., Dempsey, Angela, Britton, Jacquelyn, Kratz, Lisa, Hamosh, Ada, Vernon, Hilary, Batshaw, Mark L., Valle, David
Publicado 2023

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

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