Innovative strategies to treat protein misfolding in inborn errors of metabolism: pharmacological chaperones and proteostasis regulators
Author:
Publisher
Wiley
Subject
Genetics(clinical),Genetics
Link
http://link.springer.com/content/pdf/10.1007/s10545-014-9701-z
Reference159 articles.
1. Alfonso P, Pampin S, Estrada J et al (2005) Miglustat (NB-DNJ) works as a chaperone for mutated acid beta-glucosidase in cells transfected with several Gaucher disease mutations. Blood Cells Mol Dis 35:268–276
2. Amicus Therapeutics. (2012). Amicus Therapeutics and GlaxoSmithKline announce top line 6-month primary treatment period results from first phase 3 Fabry monotherapy study. Retrieved November 3, from http://ir.amicustherapeutics.com/releasedetail.cfm?ReleaseID=727915
3. Amicus Therapeutics. (2013). Preclinical Programs. Retrieved November 3, from http://www.amicusrx.com/preclinical.aspx
4. Anfinsen CB (1973) Principles that govern the folding of protein chains. Science 181:223–230
5. Arakawa T, Ejima D, Kita Y, Tsumoto K (2006) Small molecule pharmacological chaperones: From thermodynamic stabilization to pharmaceutical drugs. Biochim Biophys Acta 1764:1677–1687
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