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- Acute lymphoblastic leukaemia (ALL) is a rare blood cell cancer.

- It is the most common type of leukaemia in children. 

- Kymriah is an (FDA approved) gene therapy.

- This lentiviral vector delivers a special cancer-receptor gene (CAR) to immune (T) cells to make them cancer-fighting! 

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This is one example of how researchers are taking advantage of lentiviral vectors to treat disease.

 Lentiviral vectors:

  • 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 developed to cure Cystic Fibrosis!

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Other viral vectors are also being used or developed to treat disease: 

  

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Find out more about the work of the Gill Hyde Gene Medicine research group at the University of Oxford.