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Official PDF TranslationStem Cell Research & Therapy

Trehalose extricates impaired mitochondrial and autophagy dysregulation in patient iPSC-derived macular corneal dystrophy disease model

Authors: Divyani Nayak; Shivapriya Shivakumar; Rohit Shetty; K. N. Prashanthi; Arkasubhra Ghosh; Nallathambi Jeyabalan; Koushik Chakrabarty

DOI: 10.1186/s13287-024-04016-4Status: Verified Translated Edition
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Key Findings in This Report

• Patient-derived iPSC model of macular corneal dystrophy recapitulates disease-specific mitochondrial and autophagy impairments in corneal stromal keratocytes. • Trehalose treatment restores autophagic flux and mitochondrial membrane potential, reducing protein aggregates in MCD-iCSKs. • This study provides a human-relevant platform for mechanistic studies and drug screening for MCD. • Trehalose emerges as a potential non-surgical therapeutic candidate for managing macular corneal dystrophy.
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