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 - Spinal muscular atrophy (SMA) is a rare genetic condition that slowly makes muscles weaker over time, causing problems with movement.

- It's caused by a mutation in a gene known as SMN1.

- Zolgensma is an (FDA approved) gene therapy.

- This AAV9 vector delivers a functional copy of the SMN1 gene to the motor neuron cells that need it, stopping symptoms getting worse!

- This 'game-changing' gene therapy is now available on the NHS.


This is one example of how researchers are taking advantage of adeno-associated virus (AAV) vectors to treat diseases.

 Adeno-associated virus (AAV) vectors:

  • Efficient targeted delivery in vivo: many different serotypes (e.g. AAV9 used here) with different tissue or cell-type tropism.
  • Parent virus is not pathogenic in humans.
  • Long-term expression from long-lived cells.
  • Relatively easy to produce high quantities.


 Click here to discover how an AAV2 vector is also being used to cure blindness!



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.