BEAT CF
Boost the efficacy of gene therapy for Cystic Fibrosis
Ewart Kuijk, UMCU Zhiyong Lei, UMCU
Gene therapy could offer a long-term solution for people with Cystic Fibrosis (CF). However, delivering gene therapies effectively to the cells lining the airways remains a major challenge.
CF is caused by changes in the CFTR gene, leading to thick mucus and damage to the lungs and other organs. CFTR modulators can improve CFTR function for many people with CF, but these medicines are expensive and do not work for everyone.
In this public-private partnership, researchers are investigating whether medicines can make gene therapy more effective. Using miniature, lab-grown lung models called organoids, made from cells donated by people with CF, the researchers will test whether CFTR modulators can work together with gene therapy. The aim is to restore CFTR function more effectively and overcome the underlying genetic cause of CF.
What will the project do?
The project will:
* Develop gene therapy particles to restore CFTR function in lung organoids.
* Test how effectively these gene therapy particles work at different doses.
* Investigate whether combining gene therapy with CFTR modulators can improve the treatment effect.
By improving the effectiveness of gene therapy, this research could help accelerate the development of new treatments for all people with CF, including those who do not currently benefit from existing medicines.
