Adeno-associated Viruses

Adeno-associated viruses (AAVs) are small, non-pathogenic viruses that can be used as gene-therapy vectors. This means they can be used to deliver genetic material into cells to treat genetic diseases and disorders. AAVs have been used to treat a wide range of inherited diseases and have become the mainstay of gene therapy, due to their efficiency and safety compared to other virus vectors. They are particularly useful for delivering the genetic material required for gene therapy in hard-to-reach organs and tissues. AAVs are being used to develop therapeutic treatments for ailments including hemophilia, inherited retinal degeneration, limb girdle muscular dystrophy, cystic fibrosis, Huntington’s disease, and more. AAVs also have potential applications in cancer immunotherapy and vaccine delivery.