Chapter 6. Adeno-Associated Viral Vectors

Summary

Adeno-associated virus (AAV) is a non-pathogenic human virus that also has tumour suppressor properties. Its life cycle has two phases – replicative and latent. In the productive phase it co-infects the host cell with a helper virus. In the absence of a helper virus, AAV usually enters the latent phase, integrating into the human genome, commonly within a specific region of chromosome 19. Its principal advantage for gene therapy is the poor inflammatory response to infected cells. Recombinant AAV is efficient at cell entry and tends to persist in cells for long times, although expression of the transgene may be limited in both amount and duration. AAV vectors have been used in a range of cell lines and in in vitro models of specific disease applications, e.g. haemoglobinopathies and cystic fibrosis. Initial in vivo applications focused on the brain and lung as target organs.