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Targeting insulin receptor substrate-1 delays disease progression in a murine model of aniridia-associated keratopathy
Linköping University, Department of Biomedical and Clinical Sciences, Division of Sensory Organs and Communication. Linköping University, Faculty of Medicine and Health Sciences.ORCID iD: 0009-0009-7146-5167
Linköping University, Department of Biomedical and Clinical Sciences, Division of Sensory Organs and Communication. Linköping University, Faculty of Medicine and Health Sciences.ORCID iD: 0000-0003-3192-3708
Linköping University, Department of Biomedical and Clinical Sciences, Division of Sensory Organs and Communication. Linköping University, Faculty of Medicine and Health Sciences.
Linköping University, Department of Biomedical and Clinical Sciences, Division of Sensory Organs and Communication. Linköping University, Faculty of Medicine and Health Sciences.
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2025 (English)In: Biomedicine & Pharmacotherapy, ISSN 0753-3322, Vol. 191, article id 118506Article in journal (Refereed) Published
Abstract [en]

Targeting pathological corneal neovascularization arising from infection or disease is essential to preserve corneal transparency and avoid vision loss. Aniridia-associated keratopathy (AAK), a rare genetic eye condition caused by PAX6 haploinsufficiency, leads to chronic inflammation, neovascularization, and vision loss, and has limited therapeutic options. Here we evaluated Olisens®, an antisense oligonucleotide targeting insulin receptor substrate-1 (IRS-1), in a Pax6 heterozygous mouse model of AAK. Topical delivery of Olisens for 90 days significantly suppressed pathological blood and lymph vessel ingrowth into the cornea, delaying the disease course. Transcriptomic analysis revealed sustained suppression of immune responses including dendritic cell maturation, macrophage activation, and cell proliferation. Additionally, Olisens enhanced transcripts of epithelial healing factor Lars2 in the cornea and LARS2 protein expression in the peripheral epithelium and normalized peripheral corneal thickness. While vascular regrowth occurred after stopping treatment, immune pathway suppression persisted. Our results indicate targeting IRS-1 using topical Olisens reduces inflammation and neovascularization, thereby delaying AAK progression and suggesting anti-neovascular treatment as a therapeutic strategy for AAK.

Place, publisher, year, edition, pages
Elsevier BV , 2025. Vol. 191, article id 118506
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Immunology
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URN: urn:nbn:se:liu:diva-220295DOI: 10.1016/j.biopha.2025.118506PubMedID: 40885144Scopus ID: 2-s2.0-105014294773OAI: oai:DiVA.org:liu-220295DiVA, id: diva2:2026049
Projects
Angiogenesis; Aniridia-associated keratopathy; Antisense oligonucleotide; Insulin receptor substrate-1; Lars2; Lymphangiogenesis; OlisensAvailable from: 2026-01-08 Created: 2026-01-08 Last updated: 2026-01-12

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Dashti, AvaMoustardas, PetrosRautavaara, YedizzaJavidjam, DinaSefawi, IsraaLagali, Neil S
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Division of Sensory Organs and CommunicationFaculty of Medicine and Health SciencesDepartment of Ophthalmology
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1516171819202118 of 48
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