Personalized Pharmacoperones for Lysosomal Storage Disorder: Approach for Next-Generation Treatment

Advances in Protein Chemistry and Structural Biology
S A Syed Haneef, C George Priya Doss

Abstract

Lysosomal storage disorders (LSDs) are a collection of inborn errors of metabolic disorders affected by mutations in lysosome functional genes, commonly acid hydrolases. From the past decades, many approaches like enzyme replacement therapy, substrate reduction therapy are followed to treat these conditions. However, all these approaches have their own limitations. Proof-of-concept studies on pharmacological chaperone therapy (PCT) is now transformed into clinical practice to treat LSDs. Furthermore, it is narrowed with individuals to chaperone sensitive, specific mutations. Hence, personalizing the PCT will be a new direction to combat LSDs. In this review, we have discussed the available clinical strategies and pointed the light on how pharmacological chaperones can be personalized and hopeful to be a next-generation approach to address LSDs.

Citations

Nov 18, 2017·Nature Reviews. Drug Discovery·Frances M Platt
Jun 13, 2019·Current Protein & Peptide Science·Kusum YadavUpendra N Dwivedi
Mar 23, 2017·Frontiers in Pharmacology·Angie C JelinJulie Hoover-Fong
Feb 7, 2021·Annals of Neurology·Alessia Filippone, Domenico Praticò
Aug 6, 2021·Laryngo- rhino- otologie·Athanasia Warnecke, Anja Giesemann

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