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Showing 1–2 of 2 results
Advanced filters: Author: Marco Moracci Clear advanced filters

  • Pompe disease is caused by mutations in lysosomal acid α-glucosidase (GAA) and patients are being treated with recombinant human α-glucosidase (rhGAA). Here the authors present the crystal structures of rhGAA and its complexes with inhibitors and a pharmacological chaperone, which is important for drug development.

    • Véronique Roig-Zamboni
    • Beatrice Cobucci-Ponzano
    • Gerlind Sulzenbacher
    ResearchOpen Access
    Nature Communications
    Volume: 8, P: 1-10

  • The LARGE glycosyltransferase generates a repeating disaccharide on α-dystroglycan, an extracellular matrix receptor essential for muscle function. A structural study defines a unique binding mode between the LARGE-generated oligosaccharide and the matrix protein laminin.

    • David C Briggs
    • Takako Yoshida-Moriguchi
    • Kevin P Campbell
    Research
    Nature Chemical Biology
    Volume: 12, P: 810-814