Adeno-associated virus receptor (KIAA0319L) (AAVR)

Adeno-associated virus serotype 1 (AAV1) cellular entry receptors consist of a multi-step binding complex essential for the transduction of target cells. The process begins with the primary attachment of the AAV1 capsid to N-linked sialic acid moieties (alpha 2,3 and alpha 2,6 linkages) on the cell surface, which serves to concentrate the virus at the plasma membrane (Pillay et al., 2016; Wu et al., 2006). Following attachment, the virus interacts with the Adeno-associated virus receptor (AAVR), a transmembrane protein encoded by the KIAA0319L gene, which is a universal and essential receptor for most AAV serotypes, including AAV1 (Pillay et al., 2016). AAVR facilitates the internalization of the viral particle via endocytosis and its subsequent trafficking through the endosomal pathway toward the nucleus. In a clinical context, these receptors are critical for the efficacy of AAV1-based gene therapies, such as Alipogene tiparvovec, which was the first approved gene therapy in the Western world for lipoprotein lipase deficiency (Watanabe et al., 2010). Understanding the distribution and expression of these receptors is vital for predicting the tropism and safety profile of AAV1-mediated genetic medicines.

Other names

KIAA0319-like proteinAAV receptorKIAA0319LSialic acid (attachment factor)N-linked sialic acid

AAV1 utilizes N-linked sialic acid (specifically alpha 2,3 and alpha 2,6 linkages) for initial cell surface attachment, followed by interaction with the Adeno-associated virus receptor (AAVR/KIAA0319L) to facilitate endocytosis and trafficking to the nucleus for gene delivery.