Design of a Phase 3 study of AAV-mediated gene transfer of ornithine transcarbamylase (OTC) in patients with late-onset OTC deficiency

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Bibliographic Details
Published in:Molecular genetics and metabolism 2024-04, Vol.141 (4), p.108229, Article 108229
Main Authors: Konczal, Laura, Baker, Joshua, Wagenmakers, Margreet, Arnoux, Jean Baptiste, Burrow, T. Andrew, Couce, Marie Luz, del Toro, Mireia, Guelbert, Norberto, Kleppe, Soledad, Lipshutz, Gerald S., Longo, Nicola, Matsumoto, Shiro, Schulze, Andreas, Thomas, Janet, Merritt, J. Lawrence
Format: Article
Language:English
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ISSN:1096-7192
1096-7206
DOI:10.1016/j.ymgme.2024.108229