Cystic Fibrosis is the most common inherited disorder in Ireland. There are now three drugs available that can improve the lung function for most people with CF, however they do not cure the disease. But a research team in UCC is looking to change that, using a technique known as CRISPR gene editing. You may have heard of CRISPR – it won the 2020 Nobel Prize for Chemistry !
A team of researchers led by Dr. Patrick Harrison in the Department of Physiology have pioneered the application of this technique to CF for over a decade. Since their initial proof of concept study for CF gene editing in 2012, Dr Harrison’s team have successfully applied several new strategies for correcting CF mutations with greater efficiency. In the last 18 months, he has worked closely with collaborators in London, Lisbon and Paris to raise €1.7 million in research grants (€0.9 million to UCC) principally from the CF foundation (USA) and CF Trust (UK) to advance this research.
Harrison says, “Some people like to refer to CRISPR as a cutting-edge technology, as the early versions had to cut a gene in half in order to fix it. But sometimes the CF gene doesn’t join back together exactly how you want, and this limits its potential development as a drug. We’ve now identified ways to repair several CF mutations without cutting, and are now working to evaluate ways to do this for all CF mutations, and make it work efficiently in pre-clinical models”.
The Head of the College of Medicine and Health UCC, Professor Helen Whelton added;
‘’Patrick and his team in the UCC department of Physiology have been doing phenomenal work over the past number of years. The decision to focus on gene editing methods that target as many mutations as possible with the fewest number of reagents is a critical step towards clinical development and I look forward to future key findings ’’
He is also keen to point out that this work can’t be done in isolation, it requires collaboration with a huge international team, much of it facilitated by the CF Foundation’s $500 million “Path to a Cure” initiative launched in October 2019. Harrison has worked closely with the CFF since 2014, serving as an advisor to their research labs in Boston, and currently involved as co-organiser of their next gene editing workshop in 2022.
“Positive data has recently appeared in a clinical trial of gene editing of another disease for a handful of patients”, says Harrison, “We’re not there yet for CF, but we’re on the right Path”.