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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.

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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!

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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.