Start codon disruption with CRISPR/Cas9 prevents murine Fuchs' endothelial corneal dystrophy
Hironori Uehara, Xiaohui Zhang, Felipe Pereira, Siddharth Narendran, Susie Choi, Sai Bhuvanagiri, Jinlu Liu, Sangeetha Ravi Kumar, Austin D. Bohner, Lara S. Carroll, Bonnie J. Archer, Yue Zhang, Wei Liu, Guangping Gao, Jayakrishna Ambati, , Balamurali Krishna Ambati
This study shows that CRISPR/Cas9 gene editing in adult mice prevents vision loss in a model of Fuchs' endothelial corneal dystrophy by disrupting the start codon of the mutant COL8A2 gene.
eLife · Jun 8, 2021Peer-reviewedGenomics