CF is caused by mutations that abolish the function of CFTR, a protein that is needed to transport chloride and bicarbonate across cell membranes. CF mutations have been grouped in classes according to the mechanism through which they affect this protein. 10% of all CF patients with class 3 mutations can benefit from a recently approved drug, Kalydeco, which has shown to be highly effective. However, the majority of CF patients (70-90%) have different mutation (F508del) and require a different type of pharmacological treatment.
In his talk entitled "Pharmacological correction of the cystic fibrosis basic defect: problems and opportunities", Professor Luis Galietta will describe how present scientific research efforts are directed towards the identification of drugs, known as correctors, which can lessen the effects of F508del mutation. Professor Galietta and his team are among those who have been researching effective correctors by various strategies, including screenings on cultured cells of large collections of chemical compounds looking for molecules that improve the stability and function of mutant CFTR protein.
Cystic fibrosis (CF), one of the most frequent genetic diseases, is characterised by a progressive and irreversible damage of lungs, pancreas, and other organs. Ireland has the highest proportion of CF people in the world with approximately 1,200 patients suffering from this inherited chronic disease. (Source: Cystic Fibrosis Ireland)
RCSI's annual Research Day provides scientists with the opportunity to showcase their most recent research findings. The latest advances in biomedical sciences, clinical research, population health sciences, healthcare delivery and health professions education are just some of the topics featured in studies which will be presented today to more than 300 researchers who are expected to attend.
Professor Ray Stallings, Director of Research at RCSI said: ‘RCSI is committed to supporting and nurturing an active research community and the annual Research Day is a vital platform for highlighting the scope of research taking place across the different disciplines in the College. As one of Ireland's premier research institutions, exposure to world-class research begins at undergraduate level right up to the highest-calibre research-active academic staff, enabling us to train new generations of researchers and clinician scientists. We are delighted to welcome Professor Luis Galietta to share his renowned expertise in cystic fibrosis drug discovery to present his ground-breaking research with us.'
The emphasis for RCSI Research Day is on research presentations by investigators early in their career, post-doctoral fellows, post-graduate and undergraduate scholars and academic staff. All oral presentations and poster presentations will be judged and awards will be presented at a ceremony later this evening.