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A gene editing strategy for refractory CFTR mutations causing cystic fibrosis.
The goal of our project is to develop CRISPR-Cas gene editing therapy for CFTR mutations refractory to current CFTR modulators. A functional rescue will be assessed in human CF intestinal organoïds and airway cultures.
Date:3 Sep 2020 → Today
Keywords:CRISPR-Cas gene editing therapy, cystic fibrosis