AAV screen grab

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