FAQ: Using AAV for Gene Delivery

Q: What are the advantages to using AAV for gene delivery?

A: There are three main advantages to using AAV

  1. AAV has not been reported to cause any diseases.  Together with its replication defective nature, AAV has a good safety profile to be used in gene transfer in vivo and as potential gene therapy vehicles.
  2. Recombinant AAV is capable of infecting a broad range of cell types including non-dividing cells and remaining as concatemers for long-term expression.
  3. Compared with other viral vectors such as adenovirus, AAV elicits a very mild immune response in animal models.

 

Q: Can AAV be used to make stable cell lines?

A: AAV can remain in cells as concacatemers for long term expression through antibiotic selection; however permanent stable cell lines cannot be created with AAV because recombinant AAV does not integrate into the host cell genome.

 

 

Q: What are the safety requirements for AAV?

A: We recommend consulting your institution’s safety requirements for working with virus.  NIH recommends BSL2 when working with recombinant viruses, but this will vary between institutions.

 

 

Q: Can AAV be used in vivo?

A: Yes, AAV is suitable for in vivo injections. The appropriate AAV serotype should be used for expression in the tissue of interest and should be injected directly into that tissue.  For in vivo studies, AAV should be purified by either ultracentrifugation or with one of our AAV Purification Kits.