Adeno-Associated Virus _AAV_

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					       Adeno-Associated Virus (AAV)
Adeno-Associated Virus (AAV)
Risks of AAV vectors: Recent events suggest that AAV vectors may be associated with insertional
mutagenesis and cancer, and therefore, may not be as safe as previously thought. AAV can preferentially
insert at a specific site on human chromosome 19 and remain latent. Potentially at a later time when a
helper virus is present, AAV can be reactivated and produce infection. Some characteristics of AAV may
increase risk, including the following: detection in embryonic tissue, as association with male infertility,
and the ability to replicate in some cases without a helper virus.

General criteria for a risk assessment:
       Host Range: Humans and animals
       Zoonosis: AAV is not known to cause direct disease in humans.
       Clinical Manifestation: None
       Infective Dose: Unknown but can be aerosol transmitted
       Cell types able to be infected/transduced: A wide range of host of human and non-human cell
       lines cells which can be dividing and non-dividing.
       Transgene Considerations: Is the transgene oncogenic or biotoxic? If so, increased risk.
Host-vector systems
       Vector information: The long terminal repeats flank the transgene, and the AAV Rep and Cap
       and helper virus genes are provided in trans. In the absence of helper virus, recombinant AAV
       will stably integrate into the host cell genome so expression of the transgene is long term and
       stable. In humans AAV inserts preferentially at a specific site on chromosome 19.
       Helper virus: Adenovirus, herpesvirus, vaccinia virus or CMV, for replication
       Deletion of specific genes: Allows AAV to insert specifically in other chromosomal sites
General containment considerations:
       BL-1and BL-2, strain specific
Animal Research Considerations:
       Housing and husbandry: ABL-1. When a known helper virus is present or the host animal may
       potentially contain virus that could act as a helper (e.g. mice replete with retroviruses) the work
       must be done under ABL-2 containment.
       Is the animal a permissive or non-permissive host for the viral vector? Mice are permissive.
       Vector Administration: ABL-2 if a helper virus is present.
Disinfection: 10% bleach (recommended)

Contact the Division of Research Safety, Biological Safety Section (333-2755 or via e-mail at or visit our web site:

Other Biosafety Fact Sheets are available from the Biological Safety Section at our web site:

                                   Division of Research Safety
                          Phone: (217) 333-2755 • Fax: (217) 244-6594
                       E-mail: • Web site:

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