Quick Take: Adeno-Associated Virus (AAV) Vectors in Rare Diseases

The first in our series of “Quick Take” resources, this document provides an introduction to adeno-associated virus (AAV) virus vectors and their use in rare diseases.  It outlines:

1. What AAVs are
2. Why AAVs are exciting in rare diseases
3. Current and potential clinical targets for AAVs
4. Current FDA-approved therapies using AAVs
5. Clinical challenges associated with AAVs and potential solutions