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Adeno-Associated Viral (AAV) Vectors

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What are adeno-associated viral (AAV) vectors?

Most of the work done in our lab is based on the use of adeno-associated viruses (AAV) as viral vectors for gene therapy in order to express RNAs/proteins of interest. Simply, we use these viral vectors as a way to deliver our therapeutic DNA into the cells/organ of interest. Most of our vectors are produced by the UMMS Vector Core.

Why we are using adeno-associated viral (AAV) vectors

We are using AAV vectors because they work very well as a delivery tool, but most importantly they have been used in many studies in animals but also in patients where they have been shown to be safe and effective. There is even one gene therapy product approved in Europe that makes use of an AAV vector. Finally, large-scale, GMP-grade production is possible for all the serotypes that we use. That means that when we have a gene therapy product that works, it can be moved quickly to a clinical trial stage and get tested in patients.

  

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