Human stem cell-based retina on chip as new translational model for validation of AAV retinal gene therapy vectors

Gene therapies using adeno-associated viruses (AAVs) are among 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 faith...

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Main Authors: Kevin Achberger, Madalena Cipriano, Matthias Düchs, Christian Schön, Stefan Michelfelder, Birgit Stierstorfer, Thorsten Lamla, Stefan G. Kauschke, Johanna Chuchuy, Julia Roosz, Lena Mesch, Virginia Cora, Selin Pars, Natalia Pashkovskaia, Serena Corti, Sophia-Marie Hartmann, Alexander Kleger, Sebastian Kreuz, Udo Maier, Stefan Liebau, Peter Loskill
Formato: Artigo
Idioma:Inglês
Publicado em: 2021
Acesso em linha:https://doi.org/10.1016/j.stemcr.2021.08.008
https://www.cell.com/article/S2213671121004264/pdf
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