Towards a light-mediated gene therapy for the eye using caged ethinylestradiol and the inducible Cre/lox system
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 th...
Gespeichert in:
| Hauptverfasser: | , , , , , , , , , |
|---|---|
| Dokumenttyp: | Article (Journal) |
| Sprache: | Englisch |
| Veröffentlicht: |
February 26, 2024
|
| In: |
Angewandte Chemie. International edition
Year: 2024, Jahrgang: 63, Heft: 9, Pages: 1-6 |
| ISSN: | 1521-3773 |
| DOI: | 10.1002/anie.202317675 |
| Online-Zugang: | Verlag, kostenfrei, Volltext: https://doi.org/10.1002/anie.202317675 Verlag, kostenfrei, Volltext: https://onlinelibrary.wiley.com/doi/abs/10.1002/anie.202317675 
|
| Verfasserangaben: | Zoe Kiy, Juliane Chaud, Liang Xu, Eric Brandhorst, Tschackad Kamali, Carolyn Vargas, Sandro Keller, Huixiao Hong, Alexandre Specht, and Sidney Cambridge |
MARC
| LEADER | 00000caa a2200000 c 4500 | ||
|---|---|---|---|
| 001 | 1883698995 | ||
| 003 | DE-627 | ||
| 005 | 20250317130545.0 | ||
| 007 | cr uuu---uuuuu | ||
| 008 | 240318s2024 xx |||||o 00| ||eng c | ||
| 024 | 7 | |a 10.1002/anie.202317675 |2 doi | |
| 035 | |a (DE-627)1883698995 | ||
| 035 | |a (DE-599)KXP1883698995 | ||
| 035 | |a (OCoLC)1443645608 | ||
| 040 | |a DE-627 |b ger |c DE-627 |e rda | ||
| 041 | |a eng | ||
| 084 | |a 30 |2 sdnb | ||
| 100 | 1 | |a Kiy, Zoe |e VerfasserIn |0 (DE-588)1323746420 |0 (DE-627)188370751X |4 aut | |
| 245 | 1 | 0 | |a Towards a light-mediated gene therapy for the eye using caged ethinylestradiol and the inducible Cre/lox system |c Zoe Kiy, Juliane Chaud, Liang Xu, Eric Brandhorst, Tschackad Kamali, Carolyn Vargas, Sandro Keller, Huixiao Hong, Alexandre Specht, and Sidney Cambridge |
| 264 | 1 | |c February 26, 2024 | |
| 300 | |b Illustrationen | ||
| 300 | |a 6 | ||
| 336 | |a Text |b txt |2 rdacontent | ||
| 337 | |a Computermedien |b c |2 rdamedia | ||
| 338 | |a Online-Ressource |b cr |2 rdacarrier | ||
| 500 | |a Gesehen am 18.03.2024 | ||
| 500 | |a Online verfügbar: 21. Dezember 2023 | ||
| 520 | |a 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. | ||
| 650 | 4 | |a Caged Compounds | |
| 650 | 4 | |a Inducible Cre/Lox System | |
| 650 | 4 | |a Molecular Docking | |
| 650 | 4 | |a Photoactivated Transgene Expression | |
| 650 | 4 | |a Retina | |
| 700 | 1 | |a Chaud, Juliane |e VerfasserIn |4 aut | |
| 700 | 1 | |a Xu, Liang |e VerfasserIn |4 aut | |
| 700 | 1 | |a Brandhorst, Eric |d 1992- |e VerfasserIn |0 (DE-588)1360232923 |0 (DE-627)1919907165 |4 aut | |
| 700 | 1 | |a Kamali, Tschackad |e VerfasserIn |4 aut | |
| 700 | 1 | |a Vargas, Carolyn |d 1974- |e VerfasserIn |0 (DE-588)139256741 |0 (DE-627)70326740X |0 (DE-576)310920833 |4 aut | |
| 700 | 1 | |a Keller, Sandro |d 1978- |e VerfasserIn |0 (DE-588)132912996 |0 (DE-627)690990960 |0 (DE-576)299498913 |4 aut | |
| 700 | 1 | |a Hong, Huixiao |e VerfasserIn |4 aut | |
| 700 | 1 | |a Specht, Alexandre |e VerfasserIn |4 aut | |
| 700 | 1 | |a Cambridge, Sidney |e VerfasserIn |0 (DE-588)1054299390 |0 (DE-627)791264823 |0 (DE-576)410107409 |4 aut | |
| 773 | 0 | 8 | |i Enthalten in |t Angewandte Chemie. International edition |d Weinheim : Wiley-VCH, 1998 |g 63(2024), 9, Artikel-ID e202317675, Seite 1-6 |h Online-Ressource |w (DE-627)320497879 |w (DE-600)2011836-3 |w (DE-576)111552060 |x 1521-3773 |7 nnas |a Towards a light-mediated gene therapy for the eye using caged ethinylestradiol and the inducible Cre/lox system |
| 773 | 1 | 8 | |g volume:63 |g year:2024 |g number:9 |g elocationid:e202317675 |g pages:1-6 |g extent:6 |a Towards a light-mediated gene therapy for the eye using caged ethinylestradiol and the inducible Cre/lox system |
| 856 | 4 | 0 | |u https://doi.org/10.1002/anie.202317675 |x Verlag |x Resolving-System |z kostenfrei |3 Volltext |
| 856 | 4 | 0 | |u https://onlinelibrary.wiley.com/doi/abs/10.1002/anie.202317675 |x Verlag |z kostenfrei |3 Volltext |
| 951 | |a AR | ||
| 992 | |a 20240318 | ||
| 993 | |a Article | ||
| 994 | |a 2024 | ||
| 998 | |g 1323746420 |a Kiy, Zoe |m 1323746420:Kiy, Zoe |d 50000 |e 50000PK1323746420 |k 0/50000/ |p 1 |x j | ||
| 998 | |g 1360232923 |a Brandhorst, Eric |m 1360232923:Brandhorst, Eric |p 4 | ||
| 999 | |a KXP-PPN1883698995 |e 450144410X | ||
| BIB | |a Y | ||
| SER | |a journal | ||
| JSO | |a {"note":["Gesehen am 18.03.2024","Online verfügbar: 21. Dezember 2023"],"type":{"media":"Online-Ressource","bibl":"article-journal"},"recId":"1883698995","language":["eng"],"person":[{"given":"Zoe","family":"Kiy","role":"aut","roleDisplay":"VerfasserIn","display":"Kiy, Zoe"},{"given":"Juliane","family":"Chaud","role":"aut","display":"Chaud, Juliane","roleDisplay":"VerfasserIn"},{"display":"Xu, Liang","roleDisplay":"VerfasserIn","role":"aut","family":"Xu","given":"Liang"},{"display":"Brandhorst, Eric","roleDisplay":"VerfasserIn","role":"aut","family":"Brandhorst","given":"Eric"},{"given":"Tschackad","family":"Kamali","role":"aut","roleDisplay":"VerfasserIn","display":"Kamali, Tschackad"},{"given":"Carolyn","family":"Vargas","role":"aut","roleDisplay":"VerfasserIn","display":"Vargas, Carolyn"},{"role":"aut","display":"Keller, Sandro","roleDisplay":"VerfasserIn","given":"Sandro","family":"Keller"},{"display":"Hong, Huixiao","roleDisplay":"VerfasserIn","role":"aut","family":"Hong","given":"Huixiao"},{"role":"aut","display":"Specht, Alexandre","roleDisplay":"VerfasserIn","given":"Alexandre","family":"Specht"},{"family":"Cambridge","given":"Sidney","roleDisplay":"VerfasserIn","display":"Cambridge, Sidney","role":"aut"}],"title":[{"title_sort":"Towards a light-mediated gene therapy for the eye using caged ethinylestradiol and the inducible Cre/lox system","title":"Towards a light-mediated gene therapy for the eye using caged ethinylestradiol and the inducible Cre/lox system"}],"physDesc":[{"noteIll":"Illustrationen","extent":"6 S."}],"relHost":[{"disp":"Towards a light-mediated gene therapy for the eye using caged ethinylestradiol and the inducible Cre/lox systemAngewandte Chemie. International edition","note":["Gesehen am 20.12.2022","Fortsetzung der Druck-Ausgabe"],"type":{"bibl":"periodical","media":"Online-Ressource"},"language":["eng"],"corporate":[{"display":"Gesellschaft Deutscher Chemiker","roleDisplay":"Herausgebendes Organ","role":"isb"}],"recId":"320497879","pubHistory":["37.1998 -"],"titleAlt":[{"title":"Angewandte Chemie / International edition"}],"part":{"extent":"6","volume":"63","text":"63(2024), 9, Artikel-ID e202317675, Seite 1-6","issue":"9","pages":"1-6","year":"2024"},"title":[{"subtitle":"a journal of the Gesellschaft Deutscher Chemiker","title":"Angewandte Chemie","title_sort":"Angewandte Chemie","partname":"International edition"}],"physDesc":[{"extent":"Online-Ressource"}],"origin":[{"publisherPlace":"Weinheim","dateIssuedDisp":"1998-","publisher":"Wiley-VCH","dateIssuedKey":"1998"}],"id":{"zdb":["2011836-3"],"eki":["320497879"],"doi":["10.1002/(ISSN)1521-3773"],"issn":["1521-3773"]}}],"name":{"displayForm":["Zoe Kiy, Juliane Chaud, Liang Xu, Eric Brandhorst, Tschackad Kamali, Carolyn Vargas, Sandro Keller, Huixiao Hong, Alexandre Specht, and Sidney Cambridge"]},"origin":[{"dateIssuedKey":"2024","dateIssuedDisp":"February 26, 2024"}],"id":{"doi":["10.1002/anie.202317675"],"eki":["1883698995"]}} | ||
| SRT | |a KIYZOECHAUTOWARDSALI2620 | ||