rAAV-CFTRΔR rescues the cystic fibrosis phenotype in human intestinal organoids and CF mice.

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

doi:https://doi.org/10.1164/rccm.201505-0914OC

rAAV-CFTRΔR rescues the cystic fibrosis phenotype in human intestinal organoids and CF mice.

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rAAV-CFTRΔR rescues the cystic fibrosis phenotype in human intestinal organoids and CF mice.

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) American Journal of Respiratory and Critical Care Medicine, volume 193, issue 3, pp. 288 - 298

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Keywords: gene therapy, airways, patient-derived organoid cultures, viral vectors, nasal potential difference

DOI: https://doi.org/10.1164/rccm.201505-0914OC

ISSN: 1073-449X

Publisher: American Thoracic Society

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