Unlocking the Secrets of Neurodegeneration: New Potential Therapeutic Target Identified

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Unlocking the Secrets of Neurodegeneration: New Potential Therapeutic Target Identified
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Science, Space and Technology News 2024

Progressive degeneration in a neuronal network: blue represents healthy neurons, while orange and red represent the protein NPTX2. Yellow shows the toxic aggregation of the protein TDP-43. Credit: Progressive degeneration in a neuronal network: blue represents healthy neurons, while orange and red represent the protein NPTX2. Yellow shows the toxic aggregation of the protein TDP-43.

Both ALS and FTD are relentlessly progressive diseases and effective treatments are still lacking. As the population ages, the prevalence of age-related neurodegenerative diseases such as ALS and FTD is expected to increase. To mimic neurodegeneration, Marian Hruska-Plochan developed a new cell culture model called “iNets,” derived from human induced pluripotent stem cells. These cells, originated from skin cells and reprogrammed to a very early, undifferentiated stage in the laboratory, serve as a source for developing many different, desired cell types. iNets are a network of interconnected neurons and their supporting cells growing in multiple layers in a dish.

In additional experiments in the iNets model, the researchers tested whether NPTX2 could be a target for drug design to treat ALS and FTD. The team engineered a setup in which they lowered the levels of NPTX2 while neurons were suffering from TDP-43 misbehavior. They found that keeping NPTX2 levels low counteracted neurodegeneration in the iNets neurons.

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