Adeno-associated virus receptor polycystic kidney disease domain 2 (AAVR PKD2)

The AAVR PKD2 domain is the second immunoglobulin-like domain of the Adeno-associated virus receptor (AAVR), a transmembrane glycoprotein found on human cells. AAVR contains five polycystic kidney disease (PKD) domains, with PKD2 being critical for the binding and entry of multiple AAV serotypes, including AAV1, AAV2, and AAV9, but not AAV5 (which requires PKD1)[1][2][4][5][6][7]. Structural studies show that PKD2 directly interacts with the viral capsid, facilitating viral attachment and endocytosis. This interaction is central to the cellular entry pathway exploited by AAV-based gene therapy vectors, making AAVR PKD2 a key target for delivery system engineering[1][2][4][5][6][7]. There are currently no small-molecule or antibody drugs that modulate AAVR PKD2, but its interaction profile is essential for the pharmacology of gene therapeutic AAV vectors. The domain's role in mediating viral transduction also raises consideration for tissue selectivity and safety in gene therapy applications[1][7].

Other names

PKD2 domain of AAVRpolycystic kidney disease repeat domain 2 of AAVRAAVR PKD2

Gene therapy vectors utilize AAV capsid interactions with AAVR PKD2 to bind host cells, internalize, and deliver genetic cargo for therapeutic effect