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

101 Citations (Scopus)

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Medicine and Dentistry

Pharmacology, Toxicology and Pharmaceutical Science