Mutation-agnostic gene insertion therapy for RHO-associated autosomal dominant retinitis pigmentosa using zinc finger nucleases
Akishi Onishi, Tetsushi Sakuma, Michiko Mandai, Takashi Watanabe, Takaho A. Endo, Wataru Nomura, Aiko Ishimaru, Kenichi Inoue, Junki Sho, Yoko Ohigashi, Yuki Nakano, Kazushi Yasuda, Atsuta Ozaki, Akiko Maeda, Chikako Morinaga, , Masayo Takahashi
This paper develops a mutation-agnostic gene insertion therapy for RHO-adRP using ZF-ND1-mediated HITI and validates its efficacy and safety in preclinical models.
bioRxiv · Jul 17, 2026 · PreprintGenetics