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| Verfasst von: | Kiy, Zoe [VerfasserIn]  |
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| | Chaud, Juliane [VerfasserIn] |
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| | Xu, Liang [VerfasserIn] |
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| | Brandhorst, Eric [VerfasserIn] |
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| | Kamali, Tschackad [VerfasserIn] |
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| | Vargas, Carolyn [VerfasserIn] |
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| | Keller, Sandro [VerfasserIn] |
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| | Hong, Huixiao [VerfasserIn] |
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| | Specht, Alexandre [VerfasserIn] |
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| | Cambridge, Sidney [VerfasserIn] |
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| Titel: | Towards a light-mediated gene therapy for the eye using caged ethinylestradiol and the inducible Cre/lox system |
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| Verf.angabe: | Zoe Kiy, Juliane Chaud, Liang Xu, Eric Brandhorst, Tschackad Kamali, Carolyn Vargas, Sandro Keller, Huixiao Hong, Alexandre Specht, and Sidney Cambridge |
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| E-Jahr: | 2024 |
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| Jahr: | February 26, 2024 |
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| Umfang: | 6 S. |
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| Illustrationen: | Illustrationen |
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| Fussnoten: | Gesehen am 18.03.2024 ; Online verfügbar: 21. Dezember 2023 |
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| Titel Quelle: | Enthalten in: Angewandte Chemie. International edition |
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| Ort Quelle: | Weinheim : Wiley-VCH, 1998 |
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| Jahr Quelle: | 2024 |
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| Band/Heft Quelle: | 63(2024), 9, Artikel-ID e202317675, Seite 1-6 |
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| ISSN Quelle: | 1521-3773 |
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| Abstract: | Increasingly, retinal pathologies are being treated with virus-mediated gene therapies. To be able to target viral transgene expression specifically to the pathological regions of the retina with light, we established an in vivo photoactivated gene expression paradigm for retinal tissue. Based on the inducible Cre/lox system, we discovered that ethinylestradiol is a suitable alternative to Tamoxifen as ethinylestradiol is more amenable to modification with photosensitive protecting compounds, i.e., “caging.” Identification of ethinylestradiol as a ligand for the mutated human estradiol receptor was supported by in silico binding studies showing the reduced binding of caged ethinylestradiol. Caged ethinylestradiol was injected into the eyes of double transgenic GFAP-CreERT2 mice with a Cre-dependent tdTomato reporter transgene followed by irradiation with light of 450 nm. Photoactivation significantly increased retinal tdTomato expression compared to controls. We thus demonstrated a first step towards the development of a targeted, light-mediated gene therapy for the eyes. |
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| DOI: | doi:10.1002/anie.202317675 |
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| URL: | Bitte beachten Sie: Dies ist ein Bibliographieeintrag. Ein Volltextzugriff für Mitglieder der Universität besteht hier nur, falls für die entsprechende Zeitschrift/den entsprechenden Sammelband ein Abonnement besteht oder es sich um einen OpenAccess-Titel handelt.
kostenfrei: Volltext: https://doi.org/10.1002/anie.202317675 |
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| | kostenfrei: Volltext: https://onlinelibrary.wiley.com/doi/abs/10.1002/anie.202317675 |
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| | DOI: https://doi.org/10.1002/anie.202317675 |
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| Datenträger: | Online-Ressource |
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| Sprache: | eng |
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| Sach-SW: | Caged Compounds |
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| | Inducible Cre/Lox System |
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| | Molecular Docking |
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| | Photoactivated Transgene Expression |
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| | Retina |
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| K10plus-PPN: | 1883698995 |
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| Verknüpfungen: | → Zeitschrift |
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Towards a light-mediated gene therapy for the eye using caged ethinylestradiol and the inducible Cre/lox system / Kiy, Zoe [VerfasserIn]; February 26, 2024 (Online-Ressource)
