Carnitine is a pharmacological allosteric chaperone of the human lysosomal α-glucosidase

Pompe disease is an inherited metabolic disorder due to the deficiency of the lysosomal acid α-glucosidase (GAA). The only approved treatment is enzyme replacement therapy with the recombinant enzyme (rhGAA). Further approaches like pharmacological chaperone therapy, based on the stabilising effect...

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Detalles Bibliográficos
Autores principales: Iacono, Roberta, Minopoli, Nadia, Ferrara, Maria Carmina, Tarallo, Antonietta, Damiano, Carla, Porto, Caterina, Strollo, Sandra, Roig-Zamboni, Véronique, Peluso, Gianfranco, Sulzenbacher, Gerlind, Cobucci-Ponzano, Beatrice, Parenti, Giancarlo, Moracci, Marco
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Taylor & Francis 2021
Materias:
Research Paper
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8477953/
https://www.ncbi.nlm.nih.gov/pubmed/34565280
http://dx.doi.org/10.1080/14756366.2021.1975694

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8477953/
https://www.ncbi.nlm.nih.gov/pubmed/34565280
http://dx.doi.org/10.1080/14756366.2021.1975694

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