The Pharmacological Chaperone AT2220 Increases Recombinant Human Acid α-Glucosidase Uptake and Glycogen Reduction in a Mouse Model of Pompe Disease

por Khanna, Richie, Flanagan, John J., Feng, Jessie, Soska, Rebecca, Frascella, Michelle, Pellegrino, Lee J., Lun, Yi, Guillen, Darlene, Lockhart, David J., Valenzano, Kenneth J.
Publicado 2012

Co-administration With the Pharmacological Chaperone AT1001 Increases Recombinant Human α-Galactosidase A Tissue Uptake and Improves Substrate Reduction in Fabry Mice

por Benjamin, Elfrida R, Khanna, Richie, Schilling, Adriane, Flanagan, John J, Pellegrino, Lee J, Brignol, Nastry, Lun, Yi, Guillen, Darlene, Ranes, Brian E, Frascella, Michelle, Soska, Rebecca, Feng, Jessie, Dungan, Leo, Young, Brandy, Lockhart, David J, Valenzano, Kenneth J
Publicado 2012

The Pharmacological Chaperone AT2220 Increases the Specific Activity and Lysosomal Delivery of Mutant Acid Alpha-Glucosidase, and Promotes Glycogen Reduction in a Transgenic Mouse...

por Khanna, Richie, Powe, Allan C., Lun, Yi, Soska, Rebecca, Feng, Jessie, Dhulipala, Rohini, Frascella, Michelle, Garcia, Anadina, Pellegrino, Lee J., Xu, Su, Brignol, Nastry, Toth, Matthew J., Do, Hung V., Lockhart, David J., Wustman, Brandon A., Valenzano, Kenneth J.
Publicado 2014

Decreased glucocerebrosidase activity and substrate accumulation of glycosphingolipids in a novel GBA1 D409V knock-in mouse model

por Polinski, Nicole K., Martinez, Terina N., Gorodinsky, Alexander, Gareus, Ralph, Sasner, Michael, Herberth, Mark, Switzer, Robert, Ahmad, Syed O., Cosden, Mali, Kandebo, Monika, Drolet, Robert E., Buckett, Peter D., Shan, Weisong, Chen, Yi, Pellegrino, Lee J., Ellsworth, Gregory D., Dungan, Leo B., Hirst, Warren D., Clark, Sean W., Dave, Kuldip D.
Publicado 2021