Project name: Project category: Kind of research: Country: Principle investigator:

Iminosugars acting as mannosidase inhibitors in misfolded diseases as Cystic Fibrosis: Design of multivalent correctors and identification of new therapeutically targets

France Caroline Norez
Status: Ongoing

The challenge of this project is clearly 1) to propose a new pharmacological target for CF therapy and 2) to gain insights into and to better understand the mechanism of action of iminosugars-based correctors in order to generate more potent and selective correctors. Our models for misfolded proteins and human diseases include F508del-CFTR channel responsible for cystic fibrosis. The originality and novelty of this study is based first on the exploration of a new pharmaceutical target, the mannosidases of the ER, to better understand how our chemical chaperone is activating F508del-CFTR. The second novel aspect is related to the originality of the corrector structures which are based on multivalent systems displaying a controlled number of copies of mannosidase inhibitors. These compounds will be efficiently synthesized from various scaffolds by way of “click chemistry”.

Project amount (€): 20.000

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