Combining high-content imaging and repurposing of approved drugs to tackle lysosomal storage diseases

Diego L. Medina

doi:10.1117/12.2675267

12 August 2023 Combining high-content imaging and repurposing of approved drugs to tackle lysosomal storage diseases

Diego L. Medina

Proceedings Volume PC12622, Optical Methods for Inspection, Characterization, and Imaging of Biomaterials VI; PC126220N (2023) https://doi.org/10.1117/12.2675267
Event: SPIE Optical Metrology, 2023, Munich, Germany

Abstract

Lysosomal storage diseases (LSDs) are a group of genetic disorders caused by defects in lysosomal function, which lead to the accumulation of undigested substrates and subsequent cell and tissue damage. Batten disease and Mucopolysaccharidosis are two neurodegenerative LSDs that have devastating consequences for affected individuals and their families. Despite significant research efforts, there is currently no cure for these diseases. Here, we present an innovative strategy to tackle LSDs that combines high-content imaging techniques with repurposing approved drugs to identify the correctors of these diseases. Our results show that several drugs that are already approved for other indications can effectively reduce lysosomal storage in different LSDs, including batten disease and Mucopolysaccharidosis. We further validated the most promising drug candidates in mouse models of these diseases and found that they can improve several disease phenotypes, including pathological storage, motor function, and neuroinflammation. In conclusion, our findings provide a promising starting point for the development of new treatments for these devastating diseases.

Conference Presentation

Citation Download Citation

Diego L. Medina "Combining high-content imaging and repurposing of approved drugs to tackle lysosomal storage diseases", Proc. SPIE PC12622, Optical Methods for Inspection, Characterization, and Imaging of Biomaterials VI, PC126220N (12 August 2023); https://doi.org/10.1117/12.2675267

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