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

Barry J. Byrne, Shelley Collins, Cathryn Mah, Barbara Smith, Thomas Conlon, Danny Martin, Manuela Corti, Brian Cleaver, Saleem Islam, Lee Ann Lawson

Research output: Contribution to journalArticlepeer-review

32 Citations (Scopus)
Original languageEnglish
Pages (from-to)134-163
Number of pages30
JournalHuman Gene Therapy Clinical Development
Volume25
Issue number3
DOIs
Publication statusPublished - 1 Sept 2014
Externally publishedYes

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