Non-competitive pharmacologic chaperones developed through the Site-directed Enzyme Enhancement Therapy (SEE-Tx) technology for the treatment of rare genetic and neurodegenerative diseases

• Unité de recherche: INNOSUISSE
• Numéro de projet: 35449.1 IP-LS
• Titre du projet anglais: Non-competitive pharmacologic chaperones developed through the Site-directed Enzyme Enhancement Therapy (SEE-Tx) technology for the treatment of rare genetic and neurodegenerative diseases

Mots-clé (Anglais)

Life sciences, Human medicine

Résumé des résultats (Abstract) (Anglais)

In cellulo investigation of the mechanisms of action of non-competitive pharmacological chaperones that can recover misfolded mutant proteins and reduce the toxic accumulation of substrates therefore filling high unmet medical needs in the treatment of rare genetic and neurodegenerative disorders.