GeneticsbioRxivPreprint
Mutation-agnostic gene insertion therapy for RHO-associated autosomal dominant retinitis pigmentosa using zinc finger nucleases
Akishi Onishi, Tetsushi Sakuma, Michiko Mandai…·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.