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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...
Autores principales: | 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. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Public Library of Science
2012
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3427158/ https://www.ncbi.nlm.nih.gov/pubmed/22937069 http://dx.doi.org/10.1371/journal.pone.0043633 |
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