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  • Title: Personalized Pharmacoperones for Lysosomal Storage Disorder: Approach for Next-Generation Treatment.
    Author: Haneef SA, Doss CG.
    Journal: Adv Protein Chem Struct Biol; 2016; 102():225-65. PubMed ID: 26827607.
    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.
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