Dror Sharon

The paper develops an in vitro method to study how guide RNAs affect RNA editing and splicing, finding that editing can both disrupt and improve splicing outcomes depending on the target site.

This paper develops a single therapeutic molecule targeting the most common class of nonsense variants in inherited retinal diseases using anticodon-edited tRNA.

RetiGene provides a curated gene atlas for inherited retinal diseases, integrating variant data and RNA sequencing to support gene prioritization and therapeutic development.

This study identifies aberrant splicing caused by the NR2E3 c.932G>A mutation and shows that splice-shifting ASOs can restore full-length protein function.

This study fine-tunes FAM161A gene therapy in mice to restore retinal function by optimizing promoter activity, dosage, and isoform delivery.

The paper identifies INPP5E variants in non-syndromic retinal degenerations, expanding the known phenotypic spectrum of this ciliary gene.

The paper identifies pathogenic deep-intronic and structural variants in 26% of unsolved Stargardt disease cases using integrated genomics and transcriptomics.