The molecular basis for Pompe disease revealed by the structure of human acid α-glucosidase
Derrick Deming, Karen Lee, Tracey McSherry, Ronnie R. Wei, Tim Edmunds, Scott C. Garman
This paper reports the first structure of human acid α-glucosidase, revealing the molecular basis for Pompe disease and pharmacological chaperoning.
bioRxiv · Nov 1, 2017 · PreprintBiochemistry