adeno-associated virus serotype 5

Adeno-associated virus serotype 5 (AAV5) is a distinct serotype of the adeno-associated virus (AAV) family, which is a group of small, non-enveloped, single-stranded DNA viruses widely used as *vectors for gene therapy* due to their low immunogenicity, non-pathogenicity, and ability to provide long-term transgene expression in multiple tissues, including skeletal muscle, liver, and central nervous system[2][4][5]. AAV5 is notably divergent from other AAV serotypes in both genetic sequence and capsid protein structure, leading to unique tissue tropism and cellular uptake mechanisms. Unlike AAV2, which utilizes heparan sulfate for cell entry, AAV5 uses distinct cell-surface molecules and is resistant to inhibition by heparin[2]. Its unique features make it a preferred vector in some gene therapy contexts, especially where pre-existing immunity to common AAV serotypes might be limiting. AAV5-based gene therapies and diagnostics are being developed and utilized (for instance, ROCTAVIAN for hemophilia A uses an AAV5 vector; the AAV5 DetectCDx is an FDA-approved lab test detecting anti-AAV5 antibodies in patients considered for such therapies)[1][2].

Other names

adeno-associated virus type 5AAV-5AAV5AAV 5