Review
Open access
Jul 2026
Single anticodon-edited tRNA therapy targeting highly prevalent Arg>Ter premature termination codons causing inherited retinal diseases
An optimized ACE-tRNA (V3) that achieved up to 86% readthrough across 13 clinically relevant variants, restored native PRCD localization in the arRP-causing p.R22* mutant, and demonstrated activity in photoreceptor-like cells is developed.
Asodu Sandeep Sarma, Alaa Saleh, Jonathan Eintracht et al.
· bioRxiv · 0 citations