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David J. Lockhart
Researcher at Amicus Therapeutics
Publications - 76
Citations - 4173
David J. Lockhart is an academic researcher from Amicus Therapeutics. The author has contributed to research in topics: Pharmacological chaperone & Fabry disease. The author has an hindex of 25, co-authored 76 publications receiving 3911 citations. Previous affiliations of David J. Lockhart include Ambit Biosciences & National Institutes of Health.
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Journal ArticleDOI
A small molecule-kinase interaction map for clinical kinase inhibitors.
Miles A. Fabian,William H. Biggs,Daniel K. Treiber,Corey E. Atteridge,Mihai Azimioara,Mihai Azimioara,Michael G. Benedetti,Michael G. Benedetti,Todd A. Carter,Pietro Ciceri,Philip T. Edeen,Mark Floyd,Julia M. Ford,Margaret Galvin,Jay L Gerlach,Jay L Gerlach,Robert M. Grotzfeld,Sanna Herrgard,Darren E. Insko,Michael A Insko,Andiliy G. Lai,Jean-Michel Lélias,Shamal A. Mehta,Zdravko V. Milanov,Anne Marie Velasco,Lisa M. Wodicka,Hitesh K. Patel,Patrick P. Zarrinkar,David J. Lockhart +28 more
TL;DR: An efficient way to determine kinase inhibitor specificity by measuring binding of small molecules to the ATP site of kinases is described, which represents a systematic small molecule-protein interaction map for clinical compounds across a large number of related proteins.
Journal ArticleDOI
Structure of the Kinase Domain of an Imatinib-Resistant Abl Mutant in Complex with the Aurora Kinase Inhibitor VX-680
Matthew A. Young,Neil P. Shah,Luke H. Chao,Markus A. Seeliger,Zdravko V. Milanov,William H. Biggs,Daniel K. Treiber,Hitesh K. Patel,Patrick P. Zarrinkar,David J. Lockhart,Charles L. Sawyers,John Kuriyan,John Kuriyan +12 more
TL;DR: The avoidance of the innermost cavity of the Abl kinase domain by VX-680 and the specific recognition of the active conformation explain the effectiveness of this compound against mutant forms of BCR-Abl, including those with mutations at the gatekeeper position.
Journal ArticleDOI
The pharmacological chaperone 1-deoxygalactonojirimycin increases α-galactosidase A levels in Fabry patient cell lines
Elfrida R. Benjamin,John J. Flanagan,Adriane Schilling,Hui-Hwa Chang,L. Agarwal,Evan Katz,Xiaoyang Wu,C.W. Pine,Brandon W. Wustman,Robert J. Desnick,David J. Lockhart,Kenneth J. Valenzano +11 more
TL;DR: The data indicate that DGJ merits further evaluation as a treatment for patients with Fabry disease with various missense mutations, and elevated GL-3 levels in responsive Fabry fibroblasts were reduced after DGJ incubation, indicating that increased mutant α-Gal A levels can reduce accumulated substrate.
Journal ArticleDOI
The validation of pharmacogenetics for the identification of Fabry patients to be treated with migalastat.
Elfrida R. Benjamin,Maria Cecilia Della Valle,Xiaoyang Wu,Evan Katz,Farhana Pruthi,Sarah Bond,Benjamin Bronfin,Hadis Williams,Julie Yu,Daniel G. Bichet,Dominique P. Germain,Roberto Giugliani,Derralynn Hughes,Raphael Schiffmann,William R. Wilcox,Robert J. Desnick,John Kirk,Jay A. Barth,Carrolee Barlow,Kenneth J. Valenzano,Jeff Castelli,David J. Lockhart +21 more
TL;DR: The GLP HEK assay is a clinically validated method of identifying male and female Fabry patients for treatment with migalastat, a pharmacological chaperone that binds to specific mutant forms of α-galactosidase A to restore lysosomal activity.
Journal ArticleDOI
The pharmacological chaperone isofagomine increases the activity of the Gaucher disease L444P mutant form of β‐glucosidase
Richie Khanna,Elfrida R. Benjamin,Lee Pellegrino,Adriane Schilling,Brigitte Rigat,Rebecca Soska,Hadis Nafar,Brian Ranes,Jessie Feng,Yi Lun,Allan C. Powe,David Palling,Brandon Wustman,Raphael Schiffmann,Don J. Mahuran,David J. Lockhart,Kenneth J. Valenzano +16 more
TL;DR: Data suggest that IFG can increase the lysosomal activity of L444P GCase in cells and tissues, and may thus merit therapeutic evaluation for patients with neuronopathic and non‐neuronopathic Gaucher disease.