Recombinant adeno-associated virus vector

Adeno-associated virus vectors are engineered viral particles derived from wild-type AAV that have been extensively optimized for use in gene therapy as delivery vehicles[6][2][5]. These non-enveloped, single-stranded DNA viruses from the Parvoviridae family are preferred for gene transfer because they efficiently infect dividing and non-dividing cells, persist predominantly as episomal DNA (not integrated into host genome), and elicit minimal host immune response[6][5][8]. AAV vectors consist of a packaging system where the viral replication (rep) and capsid (cap) genes are replaced with an expression cassette containing the desired gene, flanked by inverted terminal repeats (ITRs)[2][6][8]. Numerous serotypes and engineered variants exist, each with unique tissue tropism determined by capsid proteins[3][4][7]. Gene transfer via AAV vectors is central to modern gene therapy applications for a wide range of diseases but, critically, **AAV vectors themselves are not molecular or pharmacological targets**; rather, they are vectors for therapeutic gene expression[8][6].

Other names

Adeno-associated virus vectorAAV vectorRecombinant AAVrAAV