Stanford Team Delivers CRISPR RNA to the Corneal Endothelium via CART Nanoparticles

Stanford University researchers Paul Wender and Howard Chang published a study in Science Advances (August 21, 2026; coverage by CRISPR Medicine News on August 24) demonstrating that biodegradable charge-altering releasable transporter (CART) nanoparticles — a non-viral RNA delivery platform Wender co-invented at Stanford — can deliver RNA selectively to the corneal endothelium, a non-regenerative cell layer whose dysfunction underlies several blinding conditions. After screening 13 CART formulations for their ability to complex RNA at acidic pH and self-degrade to release cargo, the team selected 'CART 8' and showed that two injections into the mouse anterior chamber produced edited alleles in 4.0-18.8% of posterior-cornea sequences using CRISPR/Cas9 and circular RNA cargoes. The platform also transfected human donor corneal endothelial cells in vitro and one non-human primate eye in vivo with reporter RNA, supporting feasibility for clinical translation — though the primate eye showed mild inflammation and iris transfection, and the study did not yet assess disease rescue, endothelial function, off-target editing, or repeated-dose toxicity.