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Sp2-iminosugars as chemical chaperones: a general strategy for the treatment of lysosomal storage disorders

15th National Programme for the allocation of Research Grants for Life and Matter Sciences

Rare diseases

Senior Researcher : Carmen Ortiz Mellet

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Research Centre or Institution : Facultad de Química. Universidad de Sevilla

Abstract

This project aims to explore therapeutic strategies for treating lysosomal storage disorders based on the development of compounds capable of acting as pharmacological chaperones, that is, binding strongly to the mutant protein in the endoplasmic reticulum, promoting the correct folding and facilitating transport to the lysosome. In particular, we will focus on the study of sp2 iminosugars as they have a high selectivity and are capable of adapting to the active site of different lysosomal enzymes by controlled modification of its structure. As proof of concept, it has been proposed to develop pharmacological chaperones for Gaucher's disease, Fabry disease and GM1 gangliosidosis.

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