NeurosciencebioRxivPreprint
Allele-specific correction of dominant Best vitelliform macular dystrophy in patient-derived retinal pigment epithelium
Sushma Kalmodia, Jennifer G. Aparicio, Kayla Stepanian…·Aaron Nagiel
This study corrects a dominant BEST1 mutation in patient-derived RPE cells using base editing, showing improved cellular function and transcriptome normalization.
DOI 10.64898/2026.07.09.737567