Systemic enzyme delivery by blood-brain barrier-penetrating SapC-DOPS nanovesicles for treatment of neuronopathic Gaucher disease

por Sun, Ying, Liou, Benjamin, Chu, Zhengtao, Fannin, Venette, Blackwood, Rachel, Peng, Yanyan, Grabowski, Gregory A., Davis, Harold W., Qi, Xiaoyang
Publicado 2020

Substrate Reduction Therapy Reverses Mitochondrial, mTOR, and Autophagy Alterations in a Cell Model of Gaucher Disease

por Peng, Yanyan, Liou, Benjamin, Lin, Yi, Fannin, Venette, Zhang, Wujuan, Feldman, Ricardo A., Setchell, Kenneth D. R., Grabowski, Gregory A., Sun, Ying
Publicado 2021

Combination of acid β-glucosidase mutation and Saposin C deficiency in mice reveals Gba1 mutation dependent and tissue-specific disease phenotype

por Liou, Benjamin, Zhang, Wujuan, Fannin, Venette, Quinn, Brian, Ran, Huimin, Xu, Kui, Setchell, Kenneth D. R., Witte, David, Grabowski, Gregory A., Sun, Ying
Publicado 2019

PGRN deficiency exacerbates, whereas a brain penetrant PGRN derivative protects, GBA1 mutation-associated pathologies and diseases

por Zhao, Xiangli, Lin, Yi, Liou, Benjamin, Fu, Wenyu, Jian, Jinlong, Fannin, Venette, Zhang, Wujuan, Setchell, Kenneth D. R., Grabowski, Gregory A., Sun, Ying, Liu, Chuan-ju
Publicado 2022

Biotherapy of Brain Tumors with Phosphatidylserine-Targeted Radioiodinated SapC-DOPS Nanovesicles

por Davis, Harold W., Vallabhapurapu, Subrahmanya D., Chu, Zhengtao, Wyder, Michael A., Greis, Kenneth D., Fannin, Venette, Sun, Ying, Desai, Pankaj B., Pak, Koon Y., Gray, Brian D., Qi, Xiaoyang
Publicado 2020