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adeno-associated viruses

Adeno-associated viruses (AAVs) are small, single-stranded DNA viruses that belong to the family Parvoviridae. They are characterized by their ability to infect a wide range of vertebrate hosts, including humans, without causing any known diseases. AAVs have gained significant attention in the field of gene therapy and genetic research due to their unique properties and potential applications.

Key characteristics of adeno-associated viruses include:

Nonpathogenic Nature: AAVs are considered nonpathogenic, meaning they do not typically cause diseases in their natural hosts or in humans. This attribute makes them attractive candidates for gene therapy and other medical applications.

Single-Stranded DNA: AAVs have a single-stranded DNA genome, which is relatively small (about 4.7-5.1 kilobases) compared to other DNA viruses. This genome contains the necessary genetic information for the virus to replicate and package itself.

Helper Virus Dependence: While AAVs are capable of infecting cells on their own, they require a helper virus (such as adenovirus or herpesvirus) to replicate efficiently in cells. However, in the context of gene therapy, recombinant AAVs are engineered to remove their dependency on helper viruses.

Stable Integration: AAVs are known for their ability to integrate their DNA into the host cell's genome. This feature makes them useful for gene therapy, as the inserted DNA can provide long-term expression of therapeutic genes.

Wide Tropism: Different AAV serotypes display varying tropisms (affinities) for specific cell types and tissues, allowing researchers to target specific organs or cell types for gene delivery.

Low Immunogenicity: AAVs have relatively low immunogenicity, meaning they are less likely to trigger an immune response when introduced into the body. This characteristic is advantageous for gene therapy applications.

AAVs are extensively used as vectors for gene therapy, where they are engineered to deliver therapeutic genes into target cells. These modified AAV vectors can be used to replace faulty genes, introduce beneficial genes, or modulate gene expression to treat a wide range of genetic disorders, such as inherited diseases and certain types of cancer. They have also been investigated for potential applications in vaccine development, particularly in the context of delivering viral antigens to stimulate an immune response.

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