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Analytical Chemistry|July 8, 2017
Glucosylceramide and Glucosylsphingosine Quantitation by Liquid Chromatography-Tandem Mass Spectrometry to Enable In Vivo Preclinical Studies of Neuronopathic Gaucher DiseaseRick Hamler, Nastry Brignol, Sean W Clark, et al.
Human Mutation|May 21, 2011
A pharmacogenetic approach to identify mutant forms of α-galactosidase A that respond to a pharmacological chaperone for Fabry diseaseXiaoyang Wu, Evan Katz, Maria Cecilia Della Valle, et al.
European Journal of Pharmacology|December 1, 2005
A role for cannabinoid receptors, but not endogenous opioids, in the antinociceptive activity of the CB2-selective agonist, GW405833Garth T Whiteside, Susan L Gottshall, Jamie M Boulet, et al.
The Journal of Pharmacology and Experimental Therapeutics|May 2, 2003
N-(4-tertiarybutylphenyl)-4-(3-chloropyridin-2-yl)tetrahydropyrazine -1(2H)-carbox-amide (BCTC), a novel, orally effective vanilloid receptor 1 antagonist with analgesic properties: I. in vitro characterization and pharmacokinetic propertiesKenneth J Valenzano, Elfrida R Grant, Gang Wu, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|September 24, 2009
The pharmacological chaperone 1-deoxygalactonojirimycin reduces tissue globotriaosylceramide levels in a mouse model of Fabry diseaseRichie Khanna, Rebecca Soska, Yi Lun, et al.
The Journal of Pharmacology and Experimental Therapeutics|April 1, 2004
DiPOA ([8-(3,3-diphenyl-propyl)-4-oxo-1-phenyl-1,3,8-triazaspiro[4.5]dec-3-yl]-acetic acid), a novel, systemically available, and peripherally restricted mu opioid agonist with antihyperalgesic activity: I. In vitro pharmacological characterization and pharmacokinetic propertiesKenneth J Valenzano, Wendy Miller, Zhengming Chen, et al.
The Journal of Neuroscience : the Official Journal of the Society for Neuroscience|April 13, 2012
Lysosomal dysfunction in a mouse model of Sandhoff disease leads to accumulation of ganglioside-bound amyloid-β peptideSerene Keilani, Yi Lun, Anthony C Stevens, et al.
Human Mutation|October 29, 2009
The pharmacological chaperone 1-deoxynojirimycin increases the activity and lysosomal trafficking of multiple mutant forms of acid alpha-glucosidaseJohn J Flanagan, Barbara Rossi, Katherine Tang, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|January 5, 2012
Co-administration with the pharmacological chaperone AT1001 increases recombinant human α-galactosidase A tissue uptake and improves substrate reduction in Fabry miceElfrida R Benjamin, Richie Khanna, Adriane Schilling, et al.
Plos One|July 19, 2014
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 diseaseRichie Khanna, Allan C Powe, Yi Lun, et al.
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