Engineering single-AAV CRISPR-Cas13 RNA base editors for treatment of inherited retinal diseases
Satheesh Kumar, Heng-Ai Chang, Deborah Aubin…·Guei‐Sheung Liu
This paper develops an RNA base editor using CRISPR-Cas13 and ADAR2DD to correct mutations in inherited retinal diseases, showing up to 60% editing efficiency in vitro and partial rescue in mouse models.