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

• Main Authors: Roberta Iacono (Author), Nadia Minopoli (Author), Maria Carmina Ferrara (Author), Antonietta Tarallo (Author), Carla Damiano (Author), Caterina Porto (Author), Sandra Strollo (Author), Véronique Roig-Zamboni (Author), Gianfranco Peluso (Author), Gerlind Sulzenbacher (Author), Beatrice Cobucci-Ponzano (Author), Giancarlo Parenti (Author), Marco Moracci (Author)
• Format: Book
• Published: Taylor & Francis Group, 2021-01-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...