BioengineeringbioRxivPreprint
Targeted delivery of acid alpha-glucosidase corrects skeletal muscle phenotypes in Pompe disease mice
A. D. Baik, P. T. Calafati, N. A. Aaron…·K. D. Cygnar
This study demonstrates that targeted enzyme replacement therapy using antibody-enzyme fusion proteins improves skeletal muscle phenotypes in Pompe disease mice compared to standard enzyme replacement therapy.
DOI 10.1101/2020.04.22.051672