Cystic fibrosis is caused by a mutation in the gene that encodes a particular protein, known as the cystic fibrosis transmembrane conductance regulator (or CFTR). Although this discovery was made 25 years ago and the lives of those with the disease have been extended, there is still no effective cure for the disease. Now new information about the nature of the most common form of mutation in the CFTR gene, gathered by a research team led by Dr. Gergely Lukacs of the Department of Physiology at McGill University, offers exciting new avenues for improving the treatment of the disease.
A team of researchers at the Cystic Fibrosis Translational Research Centre at McGill University and the University of British Columbia (UBC) have uncovered a new molecule for the treatment of cystic fibrosis (CF), one of the most common fatal genetic diseases. The researchers discovered the chemical from a remarkable source, a marine sponge from the South Pacific Ocean. The researchers found that the chemical corrects the localization and restores the function of the defective protein that causes CF. Their findings are published in the current issue of the journal Chemistry and Biology.