Pseudotyped AAV Vector-Mediated Gene Transfer in a Human Fetal Trachea Xenograft Model: Implications for In Utero Gene Therapy for Cystic Fibrosis

BACKGROUND: Lung disease including airway infection and inflammation currently causes the majority of morbidities and mortalities associated with cystic fibrosis (CF), making the airway epithelium and the submucosal glands (SMG) novel target cells for gene therapy in CF. These target cells are relat...

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Main Authors: Keswani, Sundeep G., Balaji, Swathi, Le, Louis, Leung, Alice, Katz, Anna B., Lim, Foong-Yen, Habli, Mounira, Jones, Helen N., Wilson, James M., Crombleholme, Timothy M.
格式: Artigo
語言:Inglês
出版: Public Library of Science 2012
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Research Article
在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC3427158/
https://ncbi.nlm.nih.gov/pubmed/22937069
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0043633
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