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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...
Autores principales: | , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
MDPI
2022
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9325019/ https://www.ncbi.nlm.nih.gov/pubmed/35890122 http://dx.doi.org/10.3390/ph15070823 |