Molecular BiologybioRxivPreprint
Cell-autonomous restoration of splicing homeostasis and RP11 phenotype in patient-derived RPE and retinal organoids by PRPF31.AAV gene therapy
Maria Elia, Valda Pauzuolyte, Μαρία Γεωργίου…·Majlinda Lako
This study demonstrates that AAV-mediated PRPF31 gene therapy restores splicing homeostasis and rescues RP11 phenotypes in patient-derived RPE and retinal organoids.
DOI 10.64898/2026.04.14.718379