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The Pharmacological Chaperone AT2220 Increases the Specific Activity and Lysosomal Delivery of Mutant Acid Alpha-Glucosidase, and Promotes Glycogen Reduction in a Transgenic Mouse Model of Pompe Disease.
Published in:
PLoS ONE, 2014, v. 9, n. 7, p. 1, doi. 10.1371/journal.pone.0102092
By:
- Khanna, Richie;
- Powe Jr, 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.
Publication type:
Article
The Pharmacological Chaperone AT2220 Increases Recombinant Human Acid α-Glucosidase Uptake and Glycogen Reduction in a Mouse Model of Pompe Disease.
Published in:
PLoS ONE, 2012, v. 7, n. 7, p. 1, doi. 10.1371/journal.pone.0040776
By:
- Khanna, Richie;
- Flanagan, John J.;
- Feng, Jessie;
- Soska, Rebecca;
- Frascella, Michelle;
- Pellegrino, Lee J.;
- Yi Lun;
- Guillen, Darlene;
- Lockhart, David J.;
- Valenzano, Kenneth J.
Publication type:
Article
Decreased glucocerebrosidase activity and substrate accumulation of glycosphingolipids in a novel GBA1 D409V knock-in mouse model.
Published in:
PLoS ONE, 2021, v. 16, n. 6, p. 1, doi. 10.1371/journal.pone.0252325
By:
- 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.
Publication type:
Article
Co-administration With the Pharmacological Chaperone AT1001 Increases Recombinant Human α-Galactosidase A Tissue Uptake and Improves Substrate Reduction in Fabry Mice.
Published in:
Molecular Therapy, 2012, v. 20, n. 4, p. 717, doi. 10.1038/mt.2011.271
By:
- 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
Publication type:
Article

Found: 4

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

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

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

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