Adeno-associated virus vector (AAV vector)

Adeno-associated virus (AAV) vectors are leading platforms for in vivo gene therapy, derived from small, non-enveloped viruses of the Parvoviridae family [1]. They are engineered to deliver therapeutic genetic material into target cells while lacking the ability to replicate or cause disease on their own [1, 4]. AAV vectors are highly valued for their ability to provide long-term transgene expression in both dividing and non-dividing cells, primarily through the formation of stable episomes in the nucleus [1]. They exhibit low genomic integration rates, which minimizes the risk of insertional mutagenesis compared to lentiviral vectors [4]. Clinically, AAV vectors are used to treat a variety of monogenic disorders, including spinal muscular atrophy and inherited retinal diseases, by replacing or supplementing a defective gene [2, 3]. Despite their success, challenges such as pre-existing host immunity (neutralizing antibodies) and high-dose-related toxicities, particularly hepatotoxicity, remain significant hurdles in their development and application [1, 4].

Other names

rAAVAdeno-associated viral vectorRecombinant adeno-associated virus

AAV vectors deliver genetic material to target cells by binding to specific cell surface receptors, undergoing endocytosis, escaping the endosome, and transporting the viral genome to the nucleus where it remains primarily as an episome for long-term transgene expression [1, 4].