Lee Pellegrino
Amicus Therapeutics (United States)(US)Genesis Medical Center(US)
Publications by Year
Research Areas
Lysosomal Storage Disorders Research, Cellular transport and secretion, Trypanosoma species research and implications, Glycogen Storage Diseases and Myoclonus, Carbohydrate Chemistry and Synthesis
Most-Cited Works
- → The pharmacological chaperone isofagomine increases the activity of the Gaucher disease L444P mutant form of β‐glucosidase(2010)158 cited
- → The Pharmacological Chaperone 1-Deoxygalactonojirimycin Reduces Tissue Globotriaosylceramide Levels in a Mouse Model of Fabry Disease(2009)138 cited
- → A GCase Chaperone Improves Motor Function in a Mouse Model of Synucleinopathy(2014)106 cited
- → Co-administration With the Pharmacological Chaperone AT1001 Increases Recombinant Human α-Galactosidase A Tissue Uptake and Improves Substrate Reduction in Fabry Mice(2012)93 cited
- → The Pharmacological Chaperone AT2220 Increases Recombinant Human Acid α-Glucosidase Uptake and Glycogen Reduction in a Mouse Model of Pompe Disease(2012)83 cited
- → Decreased glucocerebrosidase activity and substrate accumulation of glycosphingolipids in a novel GBA1 D409V knock-in mouse model(2021)40 cited
- → 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(2014)39 cited
- → Abstract 524: The novel STING agonist VB-85247 induces robust durable antitumor immune responses by intravesical administration in a non-muscle invasive bladder cancer model(2021)4 cited
- → Exploring the Use of Pharmacological Chaperone AT3375 Alone and in Combination with Recombinant human ß-Glucosidase for Gaucher Disease(2012)3 cited
- → 29. Genetic and pharmacological chaperone modulation of brain GCase activity affects synuclein accumulation in mice(2010)