Safety of Intradiaphragmatic Delivery of Adeno-Associated Virus-Mediated Alpha-Glucosidase (rAAV1-CMV-hGAA) Gene Therapy in Children Affected by Pompe Disease

Por Corti, Manuela, Liberati, Cristina, Smith, Barbara K., Lawson, Lee Ann, Tuna, Ibrahim S., Conlon, Thomas J., Coleman, Kirsten E., Islam, Saleem, Herzog, Roland W., Fuller, David D., Collins, Shelley W., Byrne, Barry J.
Publicado no Hum Gene Ther Clin Dev (2017)