Article
Topical mutant allele-specific siRNA delivery for treatment of Meesmann epithelial corneal dystrophy and elucidation of disease biomarkers.
Journal of controlled release : official journal of the Controlled Release Society - 10 Dec 2025
McLain Andrew, Saffie-Siebert Suzanne R, Nielsen Nadia Sukusu, Patterson Benjamin, Scavenius Carsten, Enghild Jan J, Marshall John, Baran-Rachwalska Paulina, Welsh Michael, Sutera Flavia M, Moore Luca, Willoughby Colin E, Nesbit M Andrew, Moore Tara
Abstract excerpt
Proven in vivo efficacy is a key requirement for novel gene therapies proceeding to clinical trial. No gene therapies for corneal dystrophies have been able to progress into patient-led studies due to a lack of suitable animal models expressing a disease phenotype comparable to that which develops in humans. Herein, we show an allele-specific siRNA targeting the L132P KRT12 mutation, causative of a severe form of...
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