Retinal cell transduction via adeno-associated virus entry mechanisms

Retinal cell transduction via adeno-associated virus (AAV) entry mechanisms refers to the cellular and molecular pathways by which AAV-based gene therapy vectors deliver their genetic payloads into retinal cells. This process requires initial binding of the AAV capsid to specific cell surface receptors, such as heparan sulfate proteoglycan (for AAV2) or sialic acid and galactose (for other AAV serotypes). Cellular proteins such as the AAV receptor (AAVR, also called KIAA0319L) are essential for post-binding uptake and intracellular transport of the viral vector. Upon endocytosis, the AAV must escape from endosomes and deliver its genome to the nucleus, leading to expression of the therapeutic gene. These mechanisms are the basis of current gene therapies for inherited retinal diseases and are critical for the development, optimization, and safety of retinal gene delivery approaches[1][4][5][6].

Other names

AAV-mediated retinal cell entryRetinal cell AAV transductionRetinal gene therapy entry mechanisms

Not applicable as a direct drug target; however, pertinent mechanisms include: Viral capsid binding to cell surface receptors (e.g., heparan sulfate proteoglycan for AAV2); Capsid–protein (AAVR) mediated endocytic uptake; Endosomal escape and nuclear entry for gene delivery