A mutation-agnostic and allele-specific ASO strategy demonstrates potent functional rescue and retinal preservation in RHO-linked retinitis pigmentosa
Salome Spaag, Wen-Hsuan Wu, Justin Yun, Aykut Demirkol, Thomas Winogrodzki, Anders Steen Knudsen, Georgy Komissarov, Chyuan‐Sheng Lin, Melita Kaltak, Maddalena Fuso, Kaushambee J Dave, Katarína Štingl, Angela Armento, Laura Kuehlewein, Britta Baumann, , Pietro De Angeli
This paper develops an allele-specific ASO strategy, SNARE, that selectively suppresses mutant RHO transcripts in RHO-linked retinitis pigmentosa, demonstrating functional rescue and retinal preservation in both in vitro and in vivo models.
bioRxiv · Sep 1, 2026 · PreprintGenetics