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Protein structural features predict responsiveness to pharmacological chaperone treatment for three lysosomal storage disorders

Three-dimensional structures of proteins can provide important clues into the efficacy of personalized treatment. We perform a structural analysis of variants within three inherited lysosomal storage disorders, comparing variants responsive to pharmacological chaperone treatment to those unresponsiv...

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Detalles Bibliográficos
Autores principales: Woodard, Jaie, Zheng, Wei, Zhang, Yang
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8478239/
https://www.ncbi.nlm.nih.gov/pubmed/34529671
http://dx.doi.org/10.1371/journal.pcbi.1009370