Journal of controlled release : official journal of the Controlled Release SocietyAnimalsRNASmall InterferingHumansBiomarkers
Topical mutant allele-specific siRNA delivery for treatment of Meesmann epithelial corneal dystrophy and elucidation of disease biomarkers.
Andrew McLain, Suzanne R Saffie-Siebert, Nadia Sukusu Nielsen, Benjamin Patterson, Carsten Scavenius, Jan J Enghild, John Marshall, Paulina Baran-Rachwalska, Michael Welsh, Flavia M Sutera, Luca Moore, Colin E Willoughby, M Andrew Nesbit, Tara Moore
Published: 202510.1016/j.jconrel.2025.114315
Abstract
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 dis…
Preview only. Read the full abstract at the source