rAAV2-CBSB-hRPE65

rAAV2-CBSB-hRPE65 is an investigational gene therapy developed for the treatment of Leber Congenital Amaurosis type 2 (LCA2), a rare inherited retinal dystrophy caused by mutations in the RPE65 gene. The therapy utilizes a recombinant adeno-associated virus serotype 2 (rAAV2) vector to deliver a functional copy of the human RPE65 cDNA to the retinal pigment epithelium (RPE) cells. Expression of the transgene is driven by a specific promoter/enhancer construct (CBSB), which typically includes a CMV enhancer and a chicken beta-actin promoter to ensure robust and stable protein production. By restoring the levels of the RPE65 protein, which is a critical isomerohydrolase in the visual cycle, the therapy aims to restore the conversion of all-trans-retinyl esters to 11-cis-retinol, thereby improving visual function and preventing further retinal degeneration in affected patients.

Other names

AAV2-hRPE65 gene therapyAAV-2-hRPE65 gene therapyAAV 2-hRPE65 gene therapyBinSheng RPE65 gene therapy