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Title

Long-Term PEDF Release in Rat Iris and Retinal Epithelial Cells after Sleeping Beauty Transposon-Mediated Gene Delivery

Authors
Garcia-Garcia, Laura
Recalde, Sergio
Hernandez, Maria
Bezunartea, Jaione
Rodriguez-Madoz, Juan Roberto
Johnen, Sandra
Diarra, Sabine
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Published in Molecular Therapy - Nucleic Acids. 2017, vol. 9, p. 1-11
Abstract Pigment epithelium derived factor (PEDF) is a potent antiangiogenic, neurotrophic, and neuroprotective molecule that is the endogenous inhibitor of vascular endothelial growth factor (VEGF) in the retina. An ex vivo gene therapy approach based on transgenic overexpression of PEDF in the eye is assumed to rebalance the angiogenic-antiangiogenic milieu of the retina, resulting in growth regression of choroidal blood vessels, the hallmark of neovascular age-related macular degeneration. Here, we show that rat pigment epithelial cells can be efficiently transfected with the PEDF-expressing non-viral hyperactive Sleeping Beauty transposon system delivered in a form free of antibiotic resistance marker miniplasmids. The engineered retinal and iris pigment epithelium cells secrete high (141 ± 13 and 222 ± 14 ng) PEDF levels in 72 hr in vitro. In vivo studies showed cell survival and insert expression during at least 4 months. Transplantation of the engineered cells to the subretinal space of a rat model of choroidal neovascularization reduces almost 50% of the development of new vessels.
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PMID: 29246287
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Article (Published version) (3.6 MB) - public document Free access
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Research group Ophtalmologie expérimentale (925)
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GARCIA-GARCIA, Laura et al. Long-Term PEDF Release in Rat Iris and Retinal Epithelial Cells after Sleeping Beauty Transposon-Mediated Gene Delivery. In: Molecular Therapy - Nucleic Acids, 2017, vol. 9, p. 1-11. https://archive-ouverte.unige.ch/unige:101755

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Deposited on : 2018-01-30

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