Engineering single-AAV CRISPR-Cas13 RNA base editors for treatment of inherited retinal diseases
Satheesh Kumar, Heng-Ai Chang, Deborah Aubin, Yi-Wen Hsiao, Alicia A. Brunet, Jun Yang, Liuhui Huang, Chi D. Luu, Alex William Hewitt, Fan Li, Lewis E Fry, Lívia S. Carvalho, Anai Gonzalez-Cordero, 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.
bioRxiv · Aug 6, 2025 · PreprintBioengineering