Single anticodon-edited tRNA therapy targeting highly prevalent Arg>Ter premature termination codons causing inherited retinal diseases
Asodu Sandeep Sarma, A Saleh, Jonathan Eintracht, Hala Kamal, Samer Khetab, Manar Salameh, Chen Matsevich, Alexey Obolensky, Eyal Banin, Dror Sharon
This paper develops a single therapeutic molecule targeting the most common class of nonsense variants in inherited retinal diseases using anticodon-edited tRNA.
bioRxiv · Jul 23, 2026 · PreprintGenetics