Lee Pellegrino

The pharmacological chaperone isofagomine increases the activity of the Gaucher disease L444P mutant form of <b>β</b>‐glucosidase

Richie Khanna, Elfrida R. Benjamin, Lee Pellegrino et al. · FEBS Journal · 2010 · 158 citations · Full text

Life Sciences, Biochemistry, Molecular Biology +16

The Pharmacological Chaperone 1-Deoxygalactonojirimycin Reduces Tissue Globotriaosylceramide Levels in a Mouse Model of Fabry Disease

Richie Khanna, Rebecca Soska, Yi Lun et al. · Molecular Therapy · 2009 · 140 citations · Full text

Life Sciences, Biochemistry, Pharmacology +16

A GCase Chaperone Improves Motor Function in a Mouse Model of Synucleinopathy

Franziska Richter, Sheila M. Fleming, Melanie B. Watson et al. · Neurotherapeutics · 2014 · 107 citations · Full text

Life Sciences, Neurobiology, Neuroscience +13

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

Elfrida R. Benjamin, Richie Khanna, Adriane Schilling et al. · Molecular Therapy · 2012 · 93 citations · Full text

Life Sciences, Biochemistry, Pharmacology +15

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

Richie Khanna, John J. Flanagan, Jessie Feng et al. · PLoS ONE · 2012 · 83 citations · Full text

Life Sciences, Biochemistry, Pharmacology +17