Cell-surface attachment factors for AAV2.5

Cell-surface attachment factors for AAV2.5 are the molecular entities on the host cell membrane that facilitate the binding and internalization of the chimeric adeno-associated virus (AAV) vector, AAV2.5. AAV2.5 is a rationally engineered capsid created by grafting five specific amino acids from AAV1 (Q263A, T265 insertion, N706A, V709A, and T717N) onto an AAV2 scaffold. This design was intended to combine the high muscle transduction efficiency of AAV1 with the well-characterized heparin-binding properties of AAV2. The primary attachment factor for AAV2.5 is Heparan Sulfate Proteoglycan (HSPG), which mediates the initial tethering of the virus to the cell surface. Following attachment, the vector utilizes the universal protein receptor AAVR (KIAA0319L) for productive entry and trafficking to the nucleus. AAV2.5 was the first engineered AAV vector to be evaluated in a clinical trial, specifically for delivering a minidystrophin gene to patients with Duchenne Muscular Dystrophy (DMD). Its efficacy and safety are closely tied to the expression of these attachment factors on target tissues and the presence of neutralizing antibodies in the patient population.

Other names

AAV2.5 receptorsAAV2.5 attachment factorsAAV2.5 entry factorsAAV2/1 chimeric receptors

Viral attachment to heparan sulfate proteoglycans followed by receptor-mediated endocytosis via AAVR