AXXAM

Aiming at Organelles as Targets for Treatment in Neurodegenerative Diseases

This work focuses on developing screening assays to study organelle dysfunction in neurodegenerative diseases such as Alzheimer’s, Parkinson’s, ALS, and frontotemporal dementia. Using disease-relevant neuronal models derived from iPSCs, the study investigates pathological phenotypes linked to lysosomal and mitochondrial dysfunction and protein aggregation.

High-content imaging, cell painting, electrophysiology on isolated lysosomes, and multielectrode array recordings were combined to characterize disease mechanisms. The assays were optimized for high-throughput screening and integrated with co-culture systems, creating a platform to model neurodegeneration and support the discovery of new therapeutic compounds.

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