AAVrh.10hCAT(KANL⁻)

AAVrh.10hCAT(KANL⁻) is an adeno-associated virus (AAV) serotype rh.10-based gene therapy designed for the treatment of dry age-related macular degeneration (AMD). Developed by researchers at Weill Cornell Medicine, the vector utilizes a CAG promoter to drive the expression of a modified human catalase (hCAT) gene. The modification involves the deletion of the C-terminal KANL sequence (amino acids 524-527), which normally serves as a peroxisomal targeting signal. By removing this sequence, the resulting catalase enzyme is secreted from the cell rather than being sequestered intracellularly. This creates a persistent extracellular antioxidant shield in the retina, neutralizing hydrogen peroxide (H2O2) and protecting retinal pigmented epithelium and photoreceptor cells from oxidative stress-induced degeneration.

Other names

AAVrh.10-CAG-hCAT(KANL⁻)