rAAV-CFTRΔR rescues the cystic fibrosis phenotype in human intestinal organoids and CF mice.
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Publication date
2016-02-01
Authors
Vidovic, D.
Carlon, MS*
da Cunha, M
Dekkers, J
Hollenhorst, M
Bijvelds, M
Ramalho, A
Van den Haute, C
Ferrante, M
Baekelandt, V
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Article
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Abstract
Keywords
gene therapy, airways, patient-derived organoid cultures, viral vectors, nasal potential difference
Citation
Vidovic, D, Carlon, MS, da Cunha, M, Dekkers, J, Hollenhorst, M, Bijvelds, M, Ramalho, A, Van den Haute, C, Ferrante, M, Baekelandt, V, Janssens, H, De Boeck, K, Sermet-Gaudelus, I, de Jonge, HR, Gijsbers, R, Beekman, J, Edelman, A & Debyser , Z 2016, 'rAAV-CFTRΔR rescues the cystic fibrosis phenotype in human intestinal organoids and CF mice.', American Journal of Respiratory and Critical Care Medicine, vol. 193 , no. 3, pp. 288-298. https://doi.org/10.1164/rccm.201505-0914OC