Biology of Adeno-Associated Virus (AAV)

One area of interest of our lab is the biology of adeno-associated virus (AAV). Adeno-associated virus is a small, nonpathogenic virus that shows great promise as a vector for gene therapy applications. AAV is a non-enveloped virus of the family Parvoviridae with a single-stranded DNA genome and an icosahedral capsid composed of the structural proteins VP1, VP2, and VP3.

AAV Biology

A particularly attractive feature of AAV is — at least in “non-dividing” tissues such as the liver and the heart — that AAV transduction results in the persistent expression of the target gene even in the absence of genome integration and, consequently, without running the risk that random insertion will result in negative consequences.

Our lab is interested in understanding the cellular mechanisms that underlie the cell and tissue specificity of AAV, mainly because of its influence on tissue tropism. The different AAV serotypes can transduce a variety of cell types and tissues, the precise nature of which depends on the specific serotype. These serotype-specific expression patterns can be explained only partially by variations in receptor densities in tissues and cell types. In contrast, it is likely that other cellular barriers are playing a role in these differences. Our goal is to improve our understanding of the cellular roadblocks that prevent the efficient transduction of certain cell types.

We are especially interested in:

  • Identification of the cellular receptors of AAV serotypes
  • Elucidation of the mechanism(s) of viral endocytosis
  • Elucidation of the molecular mechanisms of trafficking of AAV from the cell periphery to the nucleus

The viral entry and intracellular trafficking of AAV, as well as possible roadblocks to transduction, are outlined in the movie below.

Note: The images on this page are not in the public domain. The images can be used only for educational purposes.