Antisense oligonucleotide allele-specific targeting of EFEMP1 in a patient-derived model of Doyne honeycomb retinal dystrophy
Farah Olivia Rezek, Beatriz Sanchez-Pintado, Emily R. Eden, Julio C. Corral-Serrano, Nancy Aychoua, Andrew R. Webster, Thales A. C. de Guimarães, Amanda‐Jayne F. Carr, Michel Michaelides, Michael E. Cheetham, Jacqueline van der Spuy
This study develops an allele-specific antisense oligonucleotide to target EFEMP1 in a patient-derived model of Doyne honeycomb retinal dystrophy, reducing extracellular deposits and intracellular lipid accumulation.
Molecular Therapy — Nucleic Acids · May 19, 2026Peer-reviewedMolecular Biology