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Treatment of a novel CLN2 patient-derived in vitro ocular model with RGX-381 gene therapy prevents or reverses lysosomal waste accumulation

Kwi Kim,Serena Corti,Kevin Achberger,Ryan Reddinger, Chris Tully, Wei Huang,Yevgeniya Atiskova,Stefan Liebau,Angela Schulz,Simon Dulz,Peter Loskill,Nicholas Buss,Michele Fiscella,Paulo Falabella,Christina Ohnsman

INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE（2023）

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