Tripartite AAV Systems for EYS Retinal Gene Therapy
Kun-Do Rhee, Poppy Datta, Clairissa Baccam, Seongjin Seo
This paper develops a tripartite AAV system to deliver the large EYS gene using recombination, showing successful in vitro and in vivo expression of full-length EYS protein.
bioRxiv · Dec 5, 2025 · PreprintGenetics