Gene therapy to deliver cystic fibrosis transmembrane conductance regulator data

Company researchers, working with colleagues at the Howard Hughes Medical Institute, reported the results of a three-patient gene therapy protocol demonstrating that the genetic defect in CF patients could be corrected in a small part of the lining of the nose.

The study was the first to report correction of the biochemical defect associated with CF using gene therapy. Normally, the CFTR protein regulates the transport of chloride ions out of the cell. The absence of mature protein on the cell surface results in a decrease in chloride ion transport, which changes the voltage across the tissue and changes the movement of water, resulting in thick mucus secretions. ...