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Safety of Intradiaphragmatic Delivery of Adeno-Associated Virus-Mediated Alpha-Glucosidase (rAAV1-CMV- <i>hGAA</i> ) Gene Therapy in Children Affected by Pompe DiseaseManuela Corti, Barry J. Byrne, Cristina Liberati et al.|Human Gene Therapy Clinical Development|2017Cited by 118
Pompe disease gene therapyBarry J. Byrne, Cathryn Mah, Darin J. Falk et al.|Human Molecular Genetics|2011Cited by 93
Phase I/II Trial of Diaphragm Delivery of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase (rAAV1-CMV- <i>GAA</i> ) Gene Vector in Patients with Pompe DiseaseBarry J. Byrne, Shelley Collins, Barbara K. Smith et al.|Human Gene Therapy Clinical Development|2014Cited by 45
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