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

D. Vidovic, MS* Carlon, M da Cunha, J Dekkers, M Hollenhorst, M Bijvelds, A Ramalho, C Van den Haute, M Ferrante, V Baekelandt, H Janssens, K De Boeck, I Sermet-Gaudelus, HR de Jonge, R Gijsbers, J Beekman, A Edelman, Z Debyser

Research output: Contribution to journalArticleAcademicpeer-review

Original languageEnglish
Pages (from-to)288-298
JournalAmerican Journal of Respiratory and Critical Care Medicine
Volume193
Issue number3
DOIs
Publication statusPublished - 1 Feb 2016
Externally publishedYes

Keywords

  • gene therapy
  • airways
  • patient-derived organoid cultures
  • viral vectors
  • nasal potential difference

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