BiochemistrybioRxivPreprint
The molecular basis for Pompe disease revealed by the structure of human acid α-glucosidase
Derrick Deming, Karen Lee, Tracey McSherry…·Scott C. Garman
This paper reports the first structure of human acid α-glucosidase, revealing the molecular basis for Pompe disease and pharmacological chaperoning.
DOI 10.1101/212837