Adeno-associated Virus in Gene Therapy

Feb 15, 2018

Adeno-associated Virus in Gene Therapy

Adeno-associated virus (AAV) belongs to the family Parvoviridae and genus Dependovirus. Because of its non-pathogenicity, the medical field gave little to no interest at all. However, as gene therapy gains more attention, scientists are scrambling to find the best possible viral vectors for human gene therapy applications. AAV can target specific cells to correct disease-causing protein malfunction. Currently it is one of the commonly used viruses in gene therapy.


The major reasons why AAV is used in gene therapy

  1. Lack of Pathogenicity

    AAV has no known pathogenic effect in the human body. It also induces a mild immune response which makes the potential pathogenic effects negligible, making it ideal for any virus that will be used for treatment.

  2. Predictability

    AAV is relatively more predictable for its gene insertion mechanisms as compared to other types of viruses. Retroviruses, for example, have the potential of having random insertions which may cause cancer. Being predictable allows researchers to progress with gene therapy studies faster and easier.