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Projects. Life and Matter Sciences

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

Lead Researcher:
Carmen Ortiz Mellet 

Research Centre:
Facultad de Química. Universidad de Sevilla.

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.



6 articles published in Journals 
1 paper at national conferences 
2 papers at international conferences 

Carmen Ortiz Mellet  
She received her Ph.D. in Chemistry from the University of Seville in 1984, obtaining a position as a Senior Lecturer in Organic Chemistry in 1987. In 1990 she began a collaboration with Dr. Jacques Defaye's group at the Research Centre in Grenoble, France, where she conducted her research from 1990 to 1995 in the field of synthesis of complex oligosaccharide analogues and supramolecular chemistry. Since 1998 she has been the leader of the Bio-organic Chemistry of Carbohydrates Research Group at the University of Seville, reaching the post of Professor in 2008. Her current research lines have a biomedical orientation and focus on the study of carbohydrate interactions with other molecules and biomolecules and their involvement in biological and pathological processes. Among the applications being pursued are the design of glycomimetics for the treatment of lysosomal storage diseases, development of new non-viral gene vectors and the development of methodologies for the development of prebiotic and nutraceutical oligosaccharides.
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