Synthesis and characterization of fluourescent sigma-1 receptor ligands aimed at the Preclinical validation of a novel drug for treating Wolfram syndrome and Type 2A Charcot-Marie-Tooth disease
Scientific-Disciplinary Group
03/CHEM-07 - Medicinal, Toxicological, Nutraceutical-Food, Fermentation And Health And Wellbeing Products Chemistry
Description
MAMs are intracellular hotspots for: Ca2+ signaling, redox regulation, lipid exchange, mitochondrial quality control and unfolded protein response pathway. They are altered in conditions such as Wolfram syndrome (WS) or Charcot-Marie-Tooth disease Type 2 disease (CMT2A) that cause severe impairments in the quality of life (WS and CMT2A). Alteration of Ca2+ transfer from ER to mitochondria is a hallmark of WS and CMT2A. Its restoration by the activation of the sigma-1 receptor chaperone (S1R) alleviates the cellular and behavioral impairments. SIT3060 is a potent selective S1R agonist with an optimal preclinical profile. This project aims to confirm SIT3060 the compound efficacy in relevant preclinical models of WS and CMT2A, delineate the cellular mode of action on mitochondrial metabolism, generate innovative tools like quantitative fluorescent S1R ligands to demonstrate the target engagement or allow subcellular imaging, and consolidate the ADME-Tox profile.
Job posting website
https://reclutamento.ict.uniba.it/incarichi-di-ricerca/concorsi/ir-13-2026
Number of positions
1
Funding body
Università degli studi di Bari
How to apply
Other
View the original posting on the MUR website: Go to MUR website