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Page 1
A GCase chaperone improves motor function in a mouse model of synucleinopathy.
Richter F, Fleming SM, Watson M, Lemesre V, Pellegrino L, Ranes B, Zhu C, Mortazavi F, Mulligan CK, Sioshansi PC, Hean S, De La Rosa K, Khanna R, Flanagan J, Lockhart DJ, Wustman BA, Clark SW, Chesselet MF. Richter F, et al. Among authors: ranes b. Neurotherapeutics. 2014 Oct;11(4):840-56. doi: 10.1007/s13311-014-0294-x. Neurotherapeutics. 2014. PMID: 25037721 Free PMC article.
Co-administration with the pharmacological chaperone AT1001 increases recombinant human α-galactosidase A tissue uptake and improves substrate reduction in Fabry mice.
Benjamin ER, Khanna R, Schilling A, Flanagan JJ, Pellegrino LJ, Brignol N, Lun Y, Guillen D, Ranes BE, Frascella M, Soska R, Feng J, Dungan L, Young B, Lockhart DJ, Valenzano KJ. Benjamin ER, et al. Among authors: ranes be. Mol Ther. 2012 Apr;20(4):717-26. doi: 10.1038/mt.2011.271. Epub 2012 Jan 3. Mol Ther. 2012. PMID: 22215019 Free PMC article.
The pharmacological chaperone isofagomine increases the activity of the Gaucher disease L444P mutant form of beta-glucosidase.
Khanna R, Benjamin ER, Pellegrino L, Schilling A, Rigat BA, Soska R, Nafar H, Ranes BE, Feng J, Lun Y, Powe AC, Palling DJ, Wustman BA, Schiffmann R, Mahuran DJ, Lockhart DJ, Valenzano KJ. Khanna R, et al. Among authors: ranes be. FEBS J. 2010 Apr;277(7):1618-38. doi: 10.1111/j.1742-4658.2010.07588.x. Epub 2010 Feb 10. FEBS J. 2010. PMID: 20148966 Free PMC article.