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How Does AAV Work for Gene Therapy?

13 Jul 2022, 18:17 GMT+10

Adeno-associated virus (AAV), also known as a nonenveloped virus, is the most investigated vector of gene therapy. AVV has a protein shell for the protection of their genome. Earlier, protein shell was considered a contaminant of adenovirus production.

AAV is a member of the parvovirus family (small virus) and mainly depends on co-infection with adenoviruses. Firstly, it was discovered in the 1960s. The single genome of AAV contains three genes for Assembly (AAP), Replication (Rep), and capsid (Cap). The Rep gene leads to the production of four proteins of replication (Rep-40, Rep-52, Rep-68, and Rep-78). Cap encodes the production of capsid proteins (VP1, VP2, and VP3), while AAP encodes the production of assembly activating protein (AAP).

AAP has a scaffolding function of capsid assembly as it is necessary to local VP proteins. There are more than 100 AAV serotypes that vary in binding capacity of capsid protein to specific cell surface receptors. Cyagen is the well-reputed supplier of AAV vector for studying gene therapies and for field application of Cyagen knockout AAV to advance the research of patients' treatment with genetic disorders.

How Does AAV Work for Gene Therapy?

The AAV is transformed into a delivery mechanism from the naturally found virus for gene therapy. A new DNA is inserted to replace viral DNA, and AAV is a vehicle now due to the replacement of viral components. Earlier, AAV vehicle was used only to deliver normal genome to tissues or organs of the body. Nowadays, AAV is used for the delivery of therapy engineered in it. Various genetic diseases are treated through AAV technology-induced targeted therapy.

Features of AAV for Gene Therapy

1. Nonpathogenic

Studies reveal that AAV does not cause diseases when injected alone (because they cause co-infection with other viruses, mainly adenoviruses) means they are nonpathogenic.

2. Infect different and notoriously hard-to-reach tissues

The AAVs can only cause infection if another virus (adenovirus) is present because they are nonpathogenic when alone. These viruses can inject their genome into all kinds of cells with little exception. The viral surface protein molecules make them specific for the target cell and enhance local delivery.

3. Integrate into the genomes of target cells

AAV enters the human cell and integrates DNA in chromosome 19. The viral genome gets stabilized and persists with the cellular genome. The virus persistence in the human genome is described as a latent infection that can only replicate in the presence of other viruses. The viruses did not replicate there, and there is no immunogenic response against the viral genome. Such property of AVV makes it suitable for gene therapy research.

4. Hold a lot of genetic cargo

The viral genome is not very complex for experimental manipulation. The genome of various AAV strains is known due to its simple structure. The envelope protein of the virus has a unique structure that makes them specific for the selection of target cells. The specific AAV strain has a specific cellular tropism. The genetic material can be easily packed inside the viral surface protein for transfer to the target cell, and almost all cells are receptive to AAV vector DNA.

Such flexibility and inherent simplicity of AAVs make them a useful asset for advancing gene therapy.

The Applications of AAV Gene Therapy

Hereditary diseases are caused by genetic mutation and result in deficiency or malfunctioning of proteins mandatory for cellular functions. For treatment purposes, gene therapy can be performed in various ways, including defective gene replacement, silencing of the mutated gene, and overexpression of therapeutic genes. The gene silencing can be achieved by introducing hairpin RNA embedded in mRNA or via zinc finger silencing technology. All these approaches can be achieved by the most promising AAVs application.


Cyagen is supplying AAV vector for studying gene therapies and for field application of Cyagen knockout AAV to treat patients with genetic disorders. In addition, Cyagen is supplying the AAV of standard research-grade for the better-quality results of your research and patient therapies.

For more information, please visit Cyagen at

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