A cure for cystic fibrosis
- Cystic fibrosis (CF) is a genetic condition that causes sticky mucus to build up in the lungs and digestive system.
- This results in recurrent lung infection and problems with digesting food.
- Sadly the condition worsens over time.
- There are over 10,600 people living with Cystic Fibrosis in the UK alone.
- Cystic Fibrosis is caused by a mutation in a gene known as CFTR.
- Researchers here are developing a lentiviral vector to deliver a functional copy of the CFTR gene to the cells in the airway and lung that need it most!
This is one example of how researchers are taking advantage of lentiviral vectors to treat disease.
- Long-term curative potential: Integration of gene into the cell genome enables expression from daughter cells.
- Relatively large therapeutic gene capacity.
- Efficient ex vivo (outside the body) delivery.
- Can be pseudotyped (coated) with different receptors to expand tropism or enhance efficiency to target the right cells.
Click here to discover how a lentiviral vector is also being used to cure cancer!
Other viral vectors are also being used or developed to treat disease:
Find out more about the work of the Gill Hyde Gene Medicine research group at the University of Oxford.