Safety of Intradiaphragmatic Delivery of Adeno-Associated Virus-Mediated Alpha-Glucosidase (rAAV1-CMV-hGAA) Gene Therapy in Children Affected by Pompe Disease
- Manuela Corti
- , Cristina Liberati
- , Barbara K. Smith
- , Lee Ann Lawson
- , Ibrahim S. Tuna
- , Thomas J. Conlon
- , Kirsten E. Coleman
- , Saleem Islam
- , Roland W. Herzog
- , David D. Fuller
- , Shelley W. Collins
- , Barry J. Byrne
Research output: Contribution to journal › Article › peer-review
111
Citations
(Scopus)