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GCase Enhancers: A Potential Therapeutic Option for Gaucher Disease and Other Neurological Disorders

Pharmaceutical chaperones (PCs) are small compounds able to bind and stabilize misfolded proteins, allowing them to recover their native folding and thus their biological activity. In particular, lysosomal storage disorders (LSDs), a class of metabolic disorders due to genetic mutations that result...

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Autors principals: Macarena Martínez-Bailén, Francesca Clemente, Camilla Matassini, Francesca Cardona
Format: Artigo
Idioma:Inglês
Publicat: MDPI AG 2022-07-01
Col·lecció:Pharmaceuticals
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Accés en línia:https://www.mdpi.com/1424-8247/15/7/823
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