adeno-associated virus serotype 6

Adeno-associated virus serotype 6 (AAV6) is a recombinant viral vector derived from a non-enveloped parvovirus, widely utilized in gene therapy for its high transduction efficiency and specific tissue tropism. It is particularly noted for its ability to effectively target airway epithelial cells, skeletal muscle, and hematopoietic stem cells (HSCs), making it a versatile platform for treating a range of conditions including asthma, muscular disorders, and hematological diseases. AAV6 functions by delivering genetic payloads—such as transgenes for protein expression, siRNA for gene silencing, or CRISPR/Cas9 components for genome editing—into the nucleus of target cells. Its cellular entry is mediated by binding to sialic acid receptors, followed by nuclear import via the importin alpha/beta pathway. While a powerful tool for both in vivo and ex vivo applications, its clinical development involves addressing challenges such as pre-existing immunity and the potential for concatemeric vector insertions at the target site.

Other names

adeno-associated virus 6AAV-6AAV6AAV 6