Safety of Intradiaphragmatic Delivery of Adeno-Associated Virus-Mediated Alpha-Glucosidase (rAAV1-CMV-hGAA) Gene Therapy in Children Affected by Pompe Disease

A first-in-human trial of diaphragmatic gene therapy (AAV1-CMV-GAA) to treat respiratory and neural dysfunction in early-onset Pompe disease was conducted. The primary objective of this study was to assess the safety of rAAV1-CMV-hGAA vector delivered to the diaphragm muscle of Pompe disease subject...

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Detalhes bibliográficos
Publicado no:Hum Gene Ther Clin Dev
Main Authors: Corti, Manuela, Liberati, Cristina, Smith, Barbara K., Lawson, Lee Ann, Tuna, Ibrahim S., Conlon, Thomas J., Coleman, Kirsten E., Islam, Saleem, Herzog, Roland W., Fuller, David D., Collins, Shelley W., Byrne, Barry J.
Formato: Artigo
Idioma:Inglês
Publicado em: Mary Ann Liebert, Inc. 2017
Assuntos:
Research Articles
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5733674/
https://ncbi.nlm.nih.gov/pubmed/29160099
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/humc.2017.146
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