A31 MECHANISMS IN CYSTIC FIBROSIS AND OTHER BRONCHIECTATIC DISEASES: Airway Mucus Minimally Impedes Aav6 For Inhaled Lung Gene Therapy

A31 MECHANISMS IN CYSTIC FIBROSIS AND OTHER BRONCHIECTATIC DISEASES: Airway Mucus Minimally Impedes Aav6 For Inhaled Lung Gene Therapy

Viral gene vectors used in past inhaled gene therapy trials, including adenovirus and adeno-associated virus serotype 2 (AAV2), have been shown to be adhesive to human airway mucus (Hida K et al. (2014) Mol Ther. 22(8):1484-93). [...]they are likely unable to efficiently penetrate the mucus barrier...

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Veröffentlicht in:American journal of respiratory and critical care medicine 2017-01, Vol.195
Hauptverfasser: Duncan, G A, Kim, N, Colon-Cortes, Y, West, N E, Aslanidi, G, Livraghi-Butrico, A, Srivastava, A, Boucher, R C, Hanes, J, Suk, J S
Format: Artikel
Sprache:eng
Schlagworte:
Cystic fibrosis
Gene expression
Gene therapy
Lung diseases
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Zusammenfassung:Viral gene vectors used in past inhaled gene therapy trials, including adenovirus and adeno-associated virus serotype 2 (AAV2), have been shown to be adhesive to human airway mucus (Hida K et al. (2014) Mol Ther. 22(8):1484-93). [...]they are likely unable to efficiently penetrate the mucus barrier to reach the underlying epithelial cells prior to clearance.
ISSN:1073-449X
1535-4970