An Edge Hill University Associate Professor in Human Biology has helped discover a breakthrough gene therapy treatment halting the debilitating effects of Cystic Fibrosis.
Dr Aristides Tagalakis and his team have found that nanoparticles can be used in gene therapy to regulate and ‘silence’ (stop) the mutated genes responsible for producing thick mucus in the lungs of people with Cystic Fibrosis.
The life-shortening condition, which affects around 100,000 people worldwide, causes the production of thick, sticky mucus in the lungs and other organs.
Two Cystic Fibrosis patients die each week in the UK with over 90 per cent of deaths caused by thickening mucus and bacterial infections leading to inflammation and lung disease.
Currently people with the condition generally only have the option of physical (physiotherapy) and oral (drug) treatments.
But this breakthrough could lead to a permanent correction of the genes responsible providing a non-viral, long-term ‘cure’ for the condition.
Laboratory tests showed that nanoparticles can effectively penetrate mucus and silence the expression of the gene which is ‘up-regulated’ (mutated) meaning it can no longer effectively regulate mucus production.
One dose effectively silenced the gene responsible for mucus production by 30 per cent which lasted over seven days. Three doses increased the silencing to 50 per cent.
Aristides started this research at University College London with the Hart group and the work also involved professors and researchers from UCL, King’s College London and St George’s University of London who contributed to the final report.
Aristides, said: “The causative gene for Cystic Fibrosis was identified back in 1989 but this research is a major innovation. Targeted nanoparticle delivery is a new concept in Cystic Fibrosis treatment. These nanoparticles move more rapidly through mucus, can reach their target cells and can be repeatedly administered over a long period.
“The next stage is to continue this research as we hope that this nanomedicine, delivered by inhalation, will one day help children to breathe more easily and stop them from getting so many bacterial infections and often ending up in hospital.
“This could dramatically change their lives, giving them a better quality of life. Ultimately the aim of my Nanobiotechnology and Translational Medicine Group, in conjunction with Blackpool NHS Teaching Hospitals Adult Cystic Fibrosis Service (BACFS), is to use gene editing not just to silence but to permanently correct mutated genes responsible for the condition.”
The work was funded by the Cystic Fibrosis Trust, Action Medical Research and the Welcome Trust.