Intraocular siRNA Delivery Mediated by Penetratin Derivative to Silence Orthotopic Retinoblastoma Gene.

ABSTRACT: Gene therapy brings a ray of hope for inherited ocular diseases that may cause severe vision loss and even blindness. However, due to the dynamic and static absorption barriers, it is challenging to deliver genes to the posterior segment of the eye by topical instillation. To circumvent this limitation, we developed a penetratin derivative (89WP)-modified polyamidoamine polyplex to deliver small interference RNA (siRNA) via eye drops to achieve effective gene silencing in orthotopic retinoblastoma. The polyplex could be spontaneously assembled through electrostatic and hydrophobic interactions, as demonstrated by isothermal titration calorimetry, and enter cells intactly. In vitro cellular internalization revealed that the polyplex possessed higher permeability and safety than the lipoplex

SUBMITTER: Gao X 

PROVIDER: S-EPMC10053059 | biostudies-literature | 2023 Feb

REPOSITORIES: biostudies-literature