Adeno-associated virus serotype 9 vectors transduce murine alveolar and nasal epithelia and can be readministered

Abstract excerpt

Airway-directed gene transfer has emerged as a promising approach for the treatment of the two genetic diseases of the lung, namely cystic fibrosis and alpha-1-antitrypsin deficiency. Herein we describe the transduction efficiency of a novel adeno-associated virus (AAV) vector, AAV2/9, across murine nasal and lung airway epithelia. At the peak of gene expression AAV2/9-mediated human alpha-1-antitrypsin gene...

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