Kirsten E Coleman

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.

Thrombotic microangiopathy following systemic AAV administration is dependent on anti-capsid antibodies

C. Tifft, M. Corti, Precilla D’Souza, 2023, The Journal of clinical investigation.