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Use of the Novel Site-Directed Enzyme Enhancement Therapy (See-Tx) Drug Discovery Platform to Identify Pharmacological Chaperones for Glutaric Acidemia Type 1

Madalena Barroso,Alexandra Puchwein-Schwepcke,Lars Buettner, Ingrid Goebel, Katrin Kuechler,Ania C. Muntau,Aida Delgado, Ana M. Garcia-Collazo,Marc Martinell,Xavier Barril,Elena Cubero,Soren W. Gersting

JOURNAL OF MEDICINAL CHEMISTRY（2024）

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