This group of patients requires a genetic therapy approach to replace the mutated CFTR gene. We are actively working to discover and develop cystic fibrosis mRNA therapies with Moderna ...

CRISPR gene editing tools can technically locate the mutated CFTR gene, break it, and provide DNA-based instructions for the ...

This includes gene therapy, in which the mutated gene is supplemented by a healthy copy of CFTR, but the Foundation is also enthusiastic about technologies like CRISPR Cas9 to selectively correct ...

(D) CFTR: Cystic fibrosis transmembrane conductance regulator facilitates the transport of chloride ions across various cell membranes along with water (left). Mutated CFTR reduces the transport ...