Targeted approaches to induce immune tolerance for Pompe disease therapy

by Phillip A Doerfler, Sushrusha Nayak, Manuela Corti, Laurence Morel, Roland W Herzog, Barry J Byrne
Published 2016-01-01

Immune responses and hypercoagulation in ERT for Pompe disease are mutation and rhGAA dose dependent.

by Sushrusha Nayak, Phillip A Doerfler, Stacy L Porvasnik, Denise D Cloutier, Richie Khanna, Ken J Valenzano, Roland W Herzog, Barry J Byrne
Published 2014-01-01

AAV Gene Therapy Utilizing Glycosylation-Independent Lysosomal Targeting Tagged GAA in the Hypoglossal Motor System of Pompe Mice

by Brendan M. Doyle, Sara M.F. Turner, Michael D. Sunshine, Phillip A. Doerfler, Amy E. Poirier, Lauren A. Vaught, Marda L. Jorgensen, Darin J. Falk, Barry J. Byrne, David D. Fuller
Published 2019-12-01