Exosome-Associated Adeno-Associated Virus (AAV) Vector Production Services

The basic principle of gene therapy is to introduce exogenous genetic material into cells to modify, replace or regulate specific gene functions. Therefore, viral vectors represent the most promising and useful therapeutic vectors. Although virus production has been widely used in primary and applied research, new methods are still needed to improve quantity, quality, efficacy and safety, so as to narrow the gap from research results to therapeutic products.

Background of Adeno-Associated Virus

Among the viral vectors, the adeno-associated virus (AAV) vector is one of the front runners in human gene therapy because its good safety profile in clinical trials. They have shown excellent gene expression ability in rodents, non-human primates and large animal models and various human tissues. The powerful and stable gene transfer imparted by AAV to non-dividing cells is attributed to the protein capsid of the virus and the episomal configuration of its DNA in the nucleus. The tropism of AAV varies greatly between different serotypes, and advances in the use of self-complementary DNA and modified capsid technology have further expanded the efficacy of these vectors.

Overview of exo-AAV vector system. Fig.1 Overview of exo-AAV vector system. (Maguire, 2012)

Exo-AAV Vectors Development

One of the major constraints affecting their widespread use is the cost, labor, skill and time-intensive purification process of AAV. Recent studies have shown that AAV can associate with exosomes (exo-AAV) when the vector is isolated from the conditioned medium of producer cells, and the exo-AAV is more resistant to neutralizing anti-AAV antibodies than standard AAV. Compared with the classical purification scheme of iodixanol gradients of AAV, the purification of exo-AAV is actually simpler. The current purification scheme for exo-AAV isolation relies on the centrifugation step, which is to separate the cell culture supernatant containing exo-AAVs from the producer cells, and then enrich the exosomes containing AAVs.

Our Services

With the help of our advanced technology platform and the efforts of experienced scientists, Creative Biolabs provides our customers with thoughtful exo-AAV production services to help your scientific research, including:

  • AAV Vector Constructs
  • Exo-AAV Vector Preparation
  • Characterization of exo-AAV

Production diagram of Exo-AAV. Fig.2 Production diagram of Exo-AAV. (György, 2017)

In conclusion, exo-AAV appears to target cell types in a similar manner to conventionally produced AAV particles. However, exo-AAV may open up new ways to improve gene delivery. They increase transduction efficiency, expand the use of AAV in target cells and hard-to-reach areas, and are more resistant to inactivation caused by anti-AAV antibodies. If you are interested in our services, contact us or inquire us to discuss your project requirements.

References

  1. Maguire, C. A., et al. Microvesicle-associated AAV vector as a novel gene delivery system. Molecular Therapy. 2012, 20(5): 960-971.
  2. György, B., et al. Rescue of hearing by gene delivery to inner-ear hair cells using exosome-associated AAV. Molecular Therapy. 2017, 25(2): 379-391.
For Research Use Only. Cannot be used by patients.
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