Adeno-associated virus (AAV)

**Adeno-associated virus (AAV)** is a small, non-enveloped virus from the *Dependoparvovirus* genus, Parvoviridae family, containing a linear single-stranded DNA genome (~4.7-4.8 kb)[1][7]. It is replication-defective and depends on co-infection with a helper virus (adenovirus or herpesvirus) for replication[1][3][5][7]. AAVs are highly regarded as viral vectors for gene therapy because they efficiently deliver genetic material to various cell types, with minimal pathogenicity and generally low immune response[3][7]. Structural studies demonstrate an icosahedral capsid formed by 60 viral proteins (VP1/VP2/VP3), with specialized domains for cell entry and trafficking[2][3][5][6]. "Enhancement" in the AAV context relates to engineering capsid proteins, optimizing genome packaging, or employing host-cell factors or small molecules to boost infectivity, tropism, and transduction efficiency[2][4][6]. Recombinant AAVs expressing therapeutic transgenes have led to FDA-approved treatments for certain genetic diseases[6]. Safety concerns include immune responses, off-target gene delivery, and rare viral pathogenicity when co-infecting with other viruses[1][6].

Other names

AAV