Instead of animal models, organoids derived from patients' cells were used in this study.
The approach adopted by the team of the University of Trento, led by Anna Cereseto, opens new perspectives in the treatment of cystic fibrosis, a genetic disease for which no cure is currently available. The research work was carried out in collaboration with KU Leuven, in Belgium.
The research project (“A new gene therapy approach: repairing splicing mutations in the Cftr gene through genome editing”) benefited from two years funding amounting to 90,000 euro from the Italian foundation for research on cystic fibrosis (Fondazione ricerca fibrosi cistica), with the participation of the Cystic fibrosis association of Trentino (Associazione trentina fibrosi cistica). The results of the study were published today in "Nature Communications", an open access journal.
The paper will be available in Open Access at the following address: https://rdcu.be/bNoBy (The DOI for this paper will be 10.1038/s41467-019-11454-9)
More infos in the press release.