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Restoration of Aqueous Humor Outflow Following Transplantation of iPSC-Derived Trabecular Meshwork Cells in a Transgenic Mouse Model of Glaucoma
Journal article   Open access   Peer reviewed

Restoration of Aqueous Humor Outflow Following Transplantation of iPSC-Derived Trabecular Meshwork Cells in a Transgenic Mouse Model of Glaucoma

Wei Zhu, Ankur Jain, Oliver W Gramlich, Budd A Tucker, Val C Sheffield and Markus H Kuehn
Investigative ophthalmology & visual science, Vol.58(4), pp.2054-2062
04/01/2017
DOI: 10.1167/iovs.16-20672
PMCID: PMC6108236
PMID: 28384726
url
https://doi.org/10.1167/iovs.16-20672View
Published (Version of record) Open Access

Abstract

Primary open-angle glaucoma (POAG) is particularly common in older individuals and associated with pathologic degeneration of the trabecular meshwork (TM). We have shown previously that transplantation of induced pluripotent stem cell (iPSC) derived TM cells restores aqueous humor dynamics in young transgenic mice expressing a pathogenic form of human myocilin (Tg-MYOCY437H). This study was designed to determine if this approach is feasible in older mice with more pronounced TM dysfunction. Mouse iPSC were differentiated toward a TM cell phenotype (iPSC-TM) and injected into the anterior chamber of 6-month-old Tg-MYOCY437H or control mice. IOP and aqueous humor outflow facility were recorded for up to 3 months. Transmission electron microscopy, Western blot, and immunohistochemistry were performed to analyze TM morphology, quantify endoplasmic reticulum (ER) stress, and assess TM cellularity. A 12 weeks after transplantation, IOP in iPSC-TM recipients was statistically lower and outflow facility was significantly improved compared to untreated controls. The number of endogenous TM cells increased significantly in iPSC-TM recipients along with the appearance of TM cells immmunopositive for a marker of cellular division. Morphologically, transplantation of iPSC-TM preserves ER structure 12 weeks after transplantation. However, myocilin and calnexin expression levels remain elevated in transplanted eyes of these 9-month-old Tg-MYOCY437H mice, indicating that ER stress persists within the TM. Transplantation of iPSC-TM can restore IOP and outflow facility in aged Tg-MYOCY437H mice. This type of stem cell-based therapy is a promising possibility for restoration of IOP control in some glaucoma patients.
Immunohistochemistry Microscopy, Electron, Transmission Glaucoma, Open-Angle - surgery Follow-Up Studies Humans Induced Pluripotent Stem Cells - transplantation Mice, Inbred C57BL Glaucoma, Open-Angle - physiopathology Glycoproteins - metabolism Intraocular Pressure - physiology Glaucoma, Open-Angle - pathology Mice, Transgenic Induced Pluripotent Stem Cells - ultrastructure Blotting, Western Aqueous Humor - metabolism Animals Eye Proteins - metabolism Cytoskeletal Proteins - metabolism Mice Calnexin - metabolism Trabecular Meshwork - transplantation Cell Transplantation - methods Disease Models, Animal Trabecular Meshwork - cytology

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