Adeno Associated Virus Vector
What Is an Adeno-Associated Virus Vector?
Adeno-associated virus (AAV) vectors are delivery systems derived from small, non-pathogenic parvoviruses used in gene therapy (Roland W Herzog et al., 2010). Belonging to the Dependovirus genus, AAV is replication-defective and requires a helper virus, such as adenovirus, to propagate {{cite:2, 7}}. A primary safety feature is its lack of association with human disease (Michael Strauss et al., 2020). These vectors are highly valued for their ability to efficiently infect both dividing and non-dividing cells, facilitating long-term therapeutic gene expression {{cite:0, 3}}.
Structure and Composition of AAV Vectors
The AAV vector consists of an icosahedral protein capsid, approximately 18–25 nm in diameter, containing a single-stranded DNA genome of about 5 kb {{cite:0, 4, 6}}. The wild-type genome encodes Rep and Cap genes flanked by inverted terminal repeats (ITRs) (Doaa Hashad et al., 2015). In recombinant vectors, the viral genes are replaced by a transgene, while the ITRs are retained in cis to enable DNA replication and packaging into the viral particles during production {{cite:2, 5}}.
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Functional Role and Clinical Significance
AAV vectors function by delivering genetic material into the host cell nucleus, where the DNA typically forms stable episomal molecules for long-term persistence (Nancy Smyth Templeton et al., 2015). Their clinical significance stems from diverse serotypes, such as AAV2 or AAV8, which exhibit specific tissue tropisms for targeted delivery {{cite:5, 8}}. Because they induce minimal immune responses and provide high transduction efficiency in vivo, AAV vectors have become a leading platform for treating various human genetic disorders {{cite:4, 6}}.