AAV9-CAG-Gluc

Adeno-associated virus (AAV), a member of the Parvoviridae family, is widely used as a vector for gene therapy due to its safety, ability to transduce both dividing and non-dividing cells, and low immunogenicity. AAV is a small, non-enveloped, single-stranded DNA virus that has been detected in many different tissues of a variety of animals but has not been associated with any disease. Over the past decade, the discovery and development of new AAV types with significantly improved in vivo performance, unique seroreactivity, and tissue tropism have brought AAV to the forefront of gene therapy trial vector development.

AAV is a small, non-enveloped virus with a single-stranded DNA genome encapsidated in an icosahedral protein capsid. The AAV icosahedral capsid is formed by three viral proteins, VP1, VP2, and VP3, in a ratio of approximately 1:1:10. The three capsid proteins are N-terminal splice variants with a common C-terminus. VP1 is the full-length protein, VP3 is the shortest protein, and VP2 is a protein intermediate in size between VP1 and VP3. There are more than 10 AAV serotypes with capsid amino acid sequence identities of 51–99%. AAV9 has the ability to bypass the blood-brain barrier (BBB), making it the predominant capsid for transduction into the central nervous system (CNS) via systemic administration.