Allele-specific correction of dominant Best vitelliform macular dystrophy in patient-derived retinal pigment epithelium
Sushma Kalmodia, Jennifer G. Aparicio, Kayla Stepanian, Assaf Beck, ANDREW SALAS, Narine Harutyunyan, Patricia Galvan, Jinlun Bai, Maya Hayun, Meng Li, G. Esteban Fernandez, Mark W. Reid, Ryan J. Schmidt, Catherine Argyriou, David E. Cobrinik, Aaron Nagiel
This study corrects a dominant BEST1 mutation in patient-derived RPE cells using base editing, showing improved cellular function and transcriptome normalization.
bioRxiv · Jul 15, 2026 · PreprintNeuroscience