Targeted delivery of acid alpha-glucosidase corrects skeletal muscle phenotypes in Pompe disease mice
A. D. Baik, P. T. Calafati, N. A. Aaron, A. Mehra, S. Moller-Tank, L. Miloscio, L. Wang, M. Praggastis, M. S. Birnbaum, C. Pan, S. Brydges, A. O. Mujica, P. Barbounis, N. W. Gale, N. Li, , 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.
bioRxiv · Apr 22, 2020 · PreprintBioengineering