Tackling the underlying cause - Venture and Innovation Awards
Research is underway to tackle the underlying cause of cystic fibrosis (CF) in a number of ways. These studies include ways to improve the environment within the CF lung, and development of gene-based treatments to make healthy copies of the CF protein and understand how lung cells might be repaired.
Research into keeping the CF lungs healthier
Using modelling and machine learning to understand more about mucus in the CF lung.
Dr Vivek Dua, Imperial College London
Our contribution: £50,000
Partner contribution: Imperial College London, £50,000
How easy it is to clear the lungs of mucus in CF is based on the physical properties of the liquid lining the lungs, known as the air surface liquid. These physical properties include how watery, sticky and stretchy it is. Computer modelling and machine-learning approaches will be used to calculate the physical properties of the most easy-to-clear mucus. These figures can be used to design new mucus-clearing drugs in the future.
Investigating genetic treatments
Developing a gene editing method that will work for any CFTR gene mutation
Dr Patrick Harrison, University College Cork, Ireland
Our contribution: £42,000
Partner contribution: Cystic Fibrosis Foundation, £ 126,000
One way to treat cystic fibrosis is to permanently correct the damaged CFTR gene that causes the condition, known as gene editing. This technique is still under development and it is not yet available to people with CF. Most gene editing methods currently in development work by correcting specific mistakes within the gene, for example, correcting the common F508del mutation. However, Dr Harrison is developing a method of gene editing that has the potential to correct any gene mutation.
A genetic treatment approach to make healthy versions of the CF protein
Professor Stephen Hart, Institute of Child Health, UCL
Our contribution: £51,700
Partner contribution: Action Medical Research, £103,442
Professor Hart will develop a gene therapy approach that cuts out some of the more difficult steps of this process. Rather than delivering new healthy copies of the CFTR gene itself, their approach is to deliver a protein-making template of the gene to the cells. This template is called mRNA. This research project will investigate the best way to ‘package’ the mRNA to deliver it safely into the lung cells.
Developing treatments for everyone
Investigating cystic fibrosis lung repair
Dr Robert Gray, University of Edinburgh
Our contribution: £47,000
Partner contribution: Warren’s Wish Charity, £125,000
Access to CFTR modulators will not improve the pre-existing lung damage that many people with CF are living with. Within the lining of the lungs are ‘basic’ lung cells, that when activated can be converted into more specialised lung cells. Using laboratory models, researchers in Edinburgh will study whether these cells could be activated to repair the CF lung damage. This includes working out whether the repair process is likely to be hampered by having cystic fibrosis.
Research we fund
We fund research to tackle some of the most pressing issues in CF today. Find out how your donations are making a difference.
What is CF?
Cystic fibrosis, or CF, affects the lungs, digestive system and other organs, and there are over 10,600 people living with it in the UK.
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