Gel-mediated delivery of AAV1 vectors corrects ventilatory function in pompe mice with established disease

Cathryn S. Mah, Darin J. Falk, Sean A. Germain, Jeffry S. Kelley, Melissa A. Lewis, Denise A. Cloutier, Lara R. Deruisseau, Thomas J. Conlon, Kerry O. Cresawn, Thomas J. Fraites, Martha Campbell-Thompson, David D. Fuller, Barry J. Byrne