Pharmacological Chaperones: A Therapeutic Approach for Diseases Caused by Destabilizing Missense Mutations

The term “pharmacological chaperone” was introduced 20 years ago. Since then the approach with this type of drug has been proposed for several diseases, lysosomal storage disorders representing the most popular targets. The hallmark of a pharmacological chaperone is its ability to bind a protein spe...

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Vydáno v:Int J Mol Sci
Hlavní autoři: Liguori, Ludovica, Monticelli, Maria, Allocca, Mariateresa, Hay Mele, Bruno, Lukas, Jan, Cubellis, Maria Vittoria, Andreotti, Giuseppina
Médium: Artigo
Jazyk:Inglês
Vydáno: MDPI 2020
Témata:
Review
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC7014102/
https://ncbi.nlm.nih.gov/pubmed/31940970
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/ijms21020489
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