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Cystic Fibrosis Trust

Strategic Research Centre: Gene editing

Personalised engineered cell therapies for cystic fibrosis (CF).

Professor Steve Hart of UCL, gene editing lead researcherBackground

Gene editing is a new way of treating genetic diseases, including cystic fibrosis (CF). It can be used with all CF-causing mutations, and has the potential to permanently correct the affected gene. The first goal of this SRC is to use this approach to create an inhaled treatment that will fix the mutation that causes the build-up of mucus in the lungs.

The Cystic Fibrosis Trust is investing in this strategic research centre to explore the use of gene editing to fix the mutation that causes mucus to build up in the lungs of people with cystic fibrosis (CF), which is what leads to lung disease and damage to the respiratory system. Gene editing is a new and exciting way of treating many genetic diseases, including cystic fibrosis, and could offer hope to people with all variations of the genetic mutation that causes cystic fibrosis. The recent development of the CRISPR/Cas9 gene editing system has made gene editing more accessible and easily tested, and as it could provide a permanent treatment to prevent lung-related problems it is an appealing approach to investigate.

Find out more with our gene editing FAQs and a feature about our submission to a parliamentary inquiry into genome editing.

How it will work

Led by Professor Stephen Hart at University College London (rated top in the UK for research strength), this Strategic Research Centre (SRC) seeks to use gene editing, where the patient’s CF gene is precisely repaired, to correct CF-related lung problems. The study will investigate two approaches to achieve this:

First, the gene editing molecules will be wrapped in a protective coating to form 'nanoparticles' which can be inhaled and then correct the CF gene in the lung. Delivery will be repeated until enough of the patient’s cells are corrected. 

Secondly, cells will be taken from the patient and treated with gene editing in the laboratory. The patient’s own corrected cells will then be delivered back into the lungs where, once there are enough cells, the lung should now be healthy.

If one of these approaches is successful it could lead to a huge improvement in the treatment of lung disease that limits the lives of so many with cystic fibrosis.

Who is involved?

Lead Principal Investigator (PI): Professor Stephen Hart (University College London)

Co-PIs:

  • Professor Chris O’Callaghan (University College London)
  • Professor Deborah Baines (St George’s University of London)
  • Dr Adam Giangreco (University College London)
  • Dr Patrick Harrison (University College Cork)

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Research

The Trust is committed to funding and supporting cutting-edge research to find new and better treatments for cystic fibrosis. Take a look at some of that work and the progress that is being made.

Discover more

What is cystic fibrosis?

Find out more about cystic fibrosis, its diagnosis and how it is treated, as well as useful links to our publications and other organisations who can help.

Read on

FAQs

Genome editing - what is it?! You're not the only one to ask - read the answers to some frequently asked questions.

Get some answers