Recreating pathophysiology of CLN2 disease and demonstrating reversion by TPP1 gene therapy in hiPSC-derived retinal organoids and retina-on-chip

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Recreating pathophysiology of CLN2 disease and demonstrating reversion by TPP1 gene therapy in hiPSC-derived retinal organoids and retina-on-chip

Author: Corti, Serena; Kim, Kwi Hye; Chen, Ting; Botezatu, Adelina; Cora, Virginia; Ma, Ke; Pashkovskaia, Natalia; Vergara, Anamaria Bernal; Sperlich, Denise; Dave, Kaushambee; Tolone, Arianna; Reddinger, Ryan M.; Tully, Christopher B.; Higgins, Mikayla; Kleger, Alexander; Breunig, Markus; Lopatta, Paul; Wingerter, Svenja; Cipriano, Madalena; Bolz, Sylvia; Ueffing, Marius; Buss, Nicholas; Loskill, Peter; Liebau, Stefan; Achberger, Kevin
Tübinger Autor(en):
Corti, Serena
Botezatu, Adelina
Cora, Virginia
Ma, Ke
Pashkovskaia, Natalia
Vergara, Anamaria Bernal
Sperlich, Denise
Tolone, Arianna
Wingerter, Svenja
Cipriano, Madalena
Bolz, Sylvia
Ueffing, Marius
Loskill, Peter
Liebau, Stefan
Achberger, Kevin
Dave, Kaushambee J.
Published in: Cell Reports Medicine (2025), Bd. 6 (8), Article 102244
Verlagsangabe: Cambridge : Cell Press
Language: English
Full text: http://dx.doi.org/10.1016/j.xcrm.2025.102244
ISSN: 2666-3791
DDC Classifikation: 570 - Life sciences; biology
610 - Medicine and health
Dokumentart: Article
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