Research ArticleAmyotrophic Lateral Sclerosis

Genetic validation of a therapeutic target in a mouse model of ALS

Science Translational Medicine  06 Aug 2014:
Vol. 6, Issue 248, pp. 248ra104
DOI: 10.1126/scitranslmed.3009351

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A New Therapeutic Target for ALS

It has long been known that the devastating paralysis that occurs in amyotrophic lateral sclerosis (ALS) is caused by the death of motor neurons, which are responsible for communication between the brain and musculature. However, it has only more recently become appreciated that nonneuronal cells in the nervous system are altered by the disease and contribute to neuronal degeneration. Here, de Boer et al. use a combination of stem cell technologies and animal models to demonstrate that the DP1 receptor plays an important role in mediating the toxic effect that one class of nonneuronal cells, called microglia, have on motor neurons in ALS. The findings suggest that inhibiting the DP1 receptor could be a viable therapeutic strategy for treating ALS in the future.