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


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