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

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35449.1 IP-LS

Project title

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

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Key words	-	-	-
Abstract	-	-	-

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Category	Text
Key words (English)	Life sciences, Human medicine
Abstract (English)	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.