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

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

dc.contributor.author	Corti, Serena
dc.contributor.author	Botezatu, Adelina
dc.contributor.author	Cora, Virginia
dc.contributor.author	Ma, Ke
dc.contributor.author	Pashkovskaia, Natalia
dc.contributor.author	Vergara, Anamaria Bernal
dc.contributor.author	Sperlich, Denise
dc.contributor.author	Tolone, Arianna
dc.contributor.author	Wingerter, Svenja
dc.contributor.author	Cipriano, Madalena
dc.contributor.author	Bolz, Sylvia
dc.contributor.author	Ueffing, Marius
dc.contributor.author	Loskill, Peter
dc.contributor.author	Liebau, Stefan
dc.contributor.author	Achberger, Kevin
dc.contributor.author	Dave, Kaushambee J.
dc.date.accessioned	2026-05-12T06:07:52Z
dc.date.available	2026-05-12T06:07:52Z
dc.date.issued	2025
dc.identifier.issn	2666-3791
dc.identifier.uri	http://hdl.handle.net/10900/179008
dc.language.iso	en	de_DE
dc.publisher	Cambridge : Cell Press	de_DE
dc.relation.uri	http://dx.doi.org/10.1016/j.xcrm.2025.102244	de_DE
dc.subject.ddc	570	de_DE
dc.subject.ddc	610	de_DE
dc.title	Recreating pathophysiology of CLN2 disease and demonstrating reversion by TPP1 gene therapy in hiPSC-derived retinal organoids and retina-on-chip	de_DE
dc.type	Article	de_DE
utue.quellen.id	20251118000000_01033
utue.personen.roh	Corti, Serena
utue.personen.roh	Kim, Kwi Hye
utue.personen.roh	Chen, Ting
utue.personen.roh	Botezatu, Adelina
utue.personen.roh	Cora, Virginia
utue.personen.roh	Ma, Ke
utue.personen.roh	Pashkovskaia, Natalia
utue.personen.roh	Vergara, Anamaria Bernal
utue.personen.roh	Sperlich, Denise
utue.personen.roh	Dave, Kaushambee
utue.personen.roh	Tolone, Arianna
utue.personen.roh	Reddinger, Ryan M.
utue.personen.roh	Tully, Christopher B.
utue.personen.roh	Higgins, Mikayla
utue.personen.roh	Kleger, Alexander
utue.personen.roh	Breunig, Markus
utue.personen.roh	Lopatta, Paul
utue.personen.roh	Wingerter, Svenja
utue.personen.roh	Cipriano, Madalena
utue.personen.roh	Bolz, Sylvia
utue.personen.roh	Ueffing, Marius
utue.personen.roh	Buss, Nicholas
utue.personen.roh	Loskill, Peter
utue.personen.roh	Liebau, Stefan
utue.personen.roh	Achberger, Kevin
dcterms.isPartOf.ZSTitelID	Cell Reports Medicine	de_DE
dcterms.isPartOf.ZS-Issue	Article 102244	de_DE
dcterms.isPartOf.ZS-Volume	6 (8)	de_DE
utue.fakultaet	04 Medizinische Fakultät	de_DE
utue.fakultaet	07 Mathematisch-Naturwissenschaftliche Fakultät	de_DE