Johns Hopkins University Researchers Discover, Treat Toxic Effects of ALS Mutation in Neurons Using Patients’ Skin Cells

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Researchers have discovered how the most common genetic abnormality in amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) kills neurons and have successfully developed a therapeutic strategy to block this neurodegeneration in neurons made from the skin cells of ALS patients. The findings, which are published online in the October 16th issue of the Cell Press journal Neuron, have important implications for treating patients with these debilitating, currently incurable neurodegenerative diseases.

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