Molecular BiologyMolecular Therapy — Nucleic AcidsPeer-reviewed
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…·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.
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