Barbara K. Smith

Advancements in AAV-mediated Gene Therapy for Pompe Disease

B. Byrne, N. Raben, N. Clément, 2019, Journal of neuromuscular diseases.

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.

B-cell depletion is protective against anti-AAV capsid immune response: a human subject case study

T. Conlon, N. Clément, M. Corti, 2014, Molecular therapy. Methods & 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.

Adeno-associated virus-mediated gene therapy for metabolic myopathy.

B. Byrne, Angela L McCall, C. Mah, 2013, Human gene therapy.