Article

Exogenous Photoreceptor-Specific N-Glycosylated PROM1 Rescues Retinal Degeneration in Patient and Mouse Models

2025-08-07

Abstract excerpt

PROM1 is widely expressed across various tissues. However, its pathogenic mutations are exclusively associated with inherited retinal dystrophy (IRD). The mechanisms underlying this retina-specific vulnerability remain poorly understood, and no effective treatment currently exists for PROM1-IRD. Here, we utilized urine cells, hiPSCs, hiPSC-RPE cells, retinal organoids (ROs) and Prom1 -/- mice to address these ch...

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Literature Corpus work: 983b0e65-86f4-5998-b7f7-ccbbdcb3e15c
DOI: 10.1101/2025.08.06.668899

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