AAV

AAV Full Name

Adeno-associated virus

AAV Introduction

AAVs are effectively used to treat a number of genetic and acquired human diseases, more specifically, monogenic hereditary ones, i.e., diseases caused by mutations in one gene. Today, many AAV-based gene therapy medications are being investigated in preclinical and clinical trials. AAVs are small non-enveloped DNA viruses belonging to the Parvoviridae family, that were first isolated in 1965, as a contaminant in preparations of a simian adenovirus (Ad). The viruses were found incompetent to productively infect cells without a co-infection by a helper virus, usually an Ad or any type of herpesviruses, and thereby they were named as "adeno-associated", and classified into the Dependovirus genus.

Although AAVs have a high seroprevalence in humans (it has been estimated that 50% to 96% of the human population is seropositive for the second serotype of AAV (AAV2) depending on age and ethnic group), they, however, were not linked to any disease neither in humans, nor in any other species. Different AAVs have not only been detected in primates isolates, but also from avian, caprine, bovine, and equine stocks. Aside from AAV5 being the most divergent, all AAVs share a similar structure and properties. AAVs are easy to manipulate, as their particles can maintain biological stability in extreme conditions of pH and temperature. They share a genome of approximately 4.7 kb single-stranded DNA packed into an icosahedral, non-enveloped capsid with a diameter of 20–25 nm. The AAV genome consists mainly of two viral genes: rep (replication) and cap (capsid), flanked by inverted terminal repeats (ITRs).

Figure 1. Variant tropisms of AAV serotypes.
(Source: Cells. 2023)

Depending on their serotype, AAVs can have specific tropism for specific organs and tissues of the body. There are different AAV serotypes that vary in many aspects. Next, each serotype will be separately discussed. Table 1 summarizes the characteristics and properties of AAV serotypes, and Figure 1 demonstrates their variant tropisms.

Alternate Names for AAV

AAV; Adeno-associated virus

• AAV titration ELISA
• AAV titration ELISA
• Humanized AAV Antibodies
• AAV titration ELISA
• AAV titration ELISA
• AAV titration ELISA

• 1. Issa S.S., et al. Various AAV Serotypes and Their Applications in Gene Therapy: An Overview. Cells. 2023;12:785.
  2. Ling, Q., et al. AAV-based in vivo gene therapy for neurological disorders. Nat Rev Drug Discov 22. 2023, 789–806.