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Identification of Small Molecule Compounds for Pharmacological Chaperone Therapy of Aspartylglucosaminuria

Aspartylglucosaminuria (AGU) is a lysosomal storage disorder that is caused by genetic deficiency of the enzyme aspartylglucosaminidase (AGA) which is involved in glycoprotein degradation. AGU is a progressive disorder that results in severe mental retardation in early adulthood. No curative therapy...

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Detalles Bibliográficos
Autores principales:	Banning, Antje, Gülec, Christina, Rouvinen, Juha, Gray, Steven J., Tikkanen, Ritva
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Nature Publishing Group 2016
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5120323/ https://www.ncbi.nlm.nih.gov/pubmed/27876883 http://dx.doi.org/10.1038/srep37583

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