Personalized pharmacoperones for lysosomal storage disorder: approach for next-generation treatment

S. A. Syed Haneef, C. George Priya Doss

Research output: Chapter in Book/Report/Conference proceedingChapterpeer-review

13 Citations (Scopus)

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.

Original languageEnglish
Title of host publicationPersonalized medicine
EditorsRossen Donev
Place of PublicationCambridge, MA
PublisherElsevier Academic Press
Chapter8
Pages225-265
Number of pages41
Volume102
ISBN (Electronic)9780128052112
ISBN (Print)9780128047958
DOIs
Publication statusPublished - 2016
Externally publishedYes

Publication series

NameAdvances in Protein Chemistry and Structural Biology
PublisherAcademic Press Inc.
Volume102
ISSN (Print)1876-1623

Keywords

  • Enzyme Replacement Therapy/methods
  • Humans
  • Lysosomal Storage Diseases/drug therapy
  • Lysosomes/drug effects
  • Molecular Chaperones/therapeutic use
  • Pharmacogenetics
  • Precision Medicine/trends
  • Proteolysis/drug effects

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