GTB-CF-CRISPR

GTB-CF-CRISPR is an experimental CRISPR-based gene editing therapy being developed by GeneToBe for the treatment of cystic fibrosis. The program focuses on correcting mutations within the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene, which encodes a protein responsible for regulating the flow of salt and fluids in and out of cells. By utilizing CRISPR technology to repair or bypass pathogenic CFTR variants, the therapy aims to restore functional protein expression and improve clinical outcomes for patients with cystic fibrosis. The program is currently in the late discovery stage.

Other names

Cystic fibrosis CRISPR programGTB-CF-CRISPR-GeneToBe-CFTR-CRISPR-gene editing-Cystic fibrosis