This post was contributed by guest blogger Didem Goz Ayturk, a Postdoctoral Fellow in Connie Cepko’s Lab at Harvard Medical School with edits from Addgenie Karen Guerin.
Adeno-associated virus (AAV) has emerged as a favorite viral tool for both research and clinical applications. AAV can be used to transiently express a gene of interest in a variety of cell types. It was first described about 50 years ago as a contaminant of adenoviral preparations, hence the name (Atchison et al., 1965) AAV is a single stranded, DNA virus belonging to the family Parvoviridae. It has a "simple" genome packaged in an icosahedral capsid. It does not have a lipid coat, also called an envelope, and thus cannot support the addition of a glycoprotein, such as VSV-G, to its surface. In research applications, the genome is typically gutted so that precious cargo space is opened for gene delivery, and for safety. You can easily complement the virus in a tissue culture setting, in other words “in trans”, by supplying the genes that encode the replicase functions and capsid proteins. This gives researchers the ability to produce more virus in a controlled setting. Even though AAV is isolated from a wide range of organisms, it has not been associated with disease, and it is considered a biosafety level 1 (BSL1) viral agent.