title: Personalized Pharmacoperones for Lysosomal Storage Disorder: Approach for Next-Generation Treatment.
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.
authors.affiliation: Medical Biotechnology Division, School of Biosciences and Technology, VIT University, Vellore, Tamil Nadu, India.
authors.forename: S A Syed
authors.initials: SA
authors.lastname: Haneef
authors.affiliation: Medical Biotechnology Division, School of Biosciences and Technology, VIT University, Vellore, Tamil Nadu, India. Electronic address: georgecp77@yahoo.co.in.
authors.forename: C George Priya
authors.initials: CG
authors.lastname: Doss
mesh_terms: D056947:Enzyme Replacement Therapy / Q000379:methods; D006801:Humans; D016464:Lysosomal Storage Diseases / Q000188:drug therapy*; D008247:Lysosomes / Q000187:drug effects / Q000235:genetics*; D018832:Molecular Chaperones / Q000627:therapeutic use*; D010597:Pharmacogenetics; D057285:Precision Medicine / Q000639:trends*; D059748:Proteolysis / Q000187:drug effects
chemical_list: D018832:Molecular Chaperones
keywords: Combination therapy; Lysosomal storage disorders; Personalized pharmacoperones; Pharmacological chaperone therapy