Saleem Islam

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

T. Conlon, B. Byrne, I. Tuna, 2017, Human gene therapy. Clinical development.

Phase I/II trial of diaphragm delivery of recombinant adeno-associated virus acid alpha-glucosidase (rAAaV1-CMV-GAA) gene vector in patients with Pompe disease.

T. Conlon, M. Corti, S. Collins, 2014, Human gene therapy. Clinical development.