| Autor: |
Lena Mesch, Virginia Cora, Johanna Chuchuy, Stefan Michelfelder, Matthias J. Duechs, Udo Maier, Sebastian Kreuz, Stefan Kauschke, Kevin Achberger, Natalia Pashkovskaia, Madalena Cipriano, Thorsten Lamla, Stefan Liebau, Peter Loskill, C. Schoen, Serena Corti, Julia Roosz, Alexander Kleger, Birgit Stierstorfer, Selin Pars |
| Rok vydání: |
2021 |
| Předmět: |
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| DOI: |
10.1101/2021.03.02.433550 |
| Popis: |
SummaryGene therapies using adeno-associated viruses (AAVs) are amongst the most promising strategies to treat or even cure hereditary and acquired retinal diseases. However, the development of new efficient AAV vectors is slow and costly, largely because of the lack of suitable non-clinical models. By faithfully recreating structure and function of human tissues, human induced pluripotent stem cell (iPSC)-derived retinal organoids could become an essential part of the test cascade addressing translational aspects. Organ-on-Chip (OoC) technology further provides the capability to recapitulate microphysiological tissue environments as well as a precise control over structural and temporal parameters. By employing our recently developed Retina-on-chip that merges organoid and OoC-technology, we analyzed the efficacy, kinetics and cell tropism of seven first and second generation AAV vectors. The presented data demonstrate the potential of iPSC-based OoC models as the next generation of screening platforms for future gene therapeutic studies. |
| Databáze: |
OpenAIRE |
| Externí odkaz: |
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