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Adeno-associated Virus in Gene Therapy

By Esco Healthcare 5 February, 2018

 

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.

 

Proliferation of adeno-associated virus

Mass production of viral vectors have become an important factor for gene therapy. The Tide Motion technology is excellent for AAV production which requires transient transfection of adenovirus in host cells such as HEK-293. For example, the Hybrid bioreactor can generate AAVs by proliferating host cells at a high density and infecting these cells afterwards. During this process, cells will be attached to 3D macrocarriers (BioNOC™II), which has better porosity for oxygen and nutrition uptake and lower lint generation. Once confluence is reached, AAVs will be transfected in the culture medium through perfusion. AAVs will then deliver their DNA into the nucleus of the cell for a robust recombinant antibody production. Large amounts of these vectors will be found in the culture supernatant, which can then be continuously harvested from the bioreactor. After harvesting, new culture medium can perfuse in the bioreactor for continuous production. This 100% media exchange is a process that is favorable in AAV vector production. Flexibility in terms of purification after culture is also reduced since BioNOC™ II generates low lint waste. This resolves the time-consuming, downstream processing issue.

Tide Motion bioreactors are linearly scalable from laboratory to production scale, thus eliminating the scale-up problem in producing AAVs. Technology transfer from roller bottles or other 2D culture methods to Tide Motion bioreactors also provide a significant indication that same bioprocessing parameters can be utilized for an efficient process. The mechanism of AAV DNA persistence have been taken to improve more on the AAV vector production. With this, utilizing Tide Motion bioreactors for AAV culture satisfy most of the requirement needed for inherent advantage in process speed and product yield.

 

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About Adeno-associated Virus (AAV)

Adeno-associated virus are small viruses from the Parvovirus family that can insert genetic material at a specific site of a chromosome. It is uniquely suited for gene therapy application since it cannot replicate and spread within the body.

Source: http://www.genetherapynet.com/viral-vector/adeno-associated-viruses.html

 

About Tide Motion Bioreactors

Tide Motion pertains to the gentle oscillation of culture medium into and out of the matrix vessel that intermittently exposes the cells to aeration and nutrition. The upward oscillation exposes the cells to nutrition, while the downward oscillation exposes the cells to aeration. At the same time, this process washes away products and wastes. This oscillation produces no air bubbles and low shear stress. View a range of products at http://www.vaccixcell.com/tide-technology/

 

About Esco VacciXcell

Esco VacciXcell is the bioprocessing division of Esco Group of Companies that specializes in the marketing and manufacturing of bioprocessing equipment for cell culture.

Esco VacciXcell provides turnkey manufacturing solutions using its proprietary Tide Motion technology to help developing nations to be self-sufficient in the manufacturing, storing, distribution, and administration of vaccines and other biologics, thus providing a complete solution from Discovery to Delivery. For more information on VacciXcell, please visit www.vaccixcell.com.