Timothy Miller, MD, PhD
Washington University in St. Louis
miRNAs enriched in ALS-vulnerable motor neurons as therapeutic targets
Cells in the spinal cord known as microglia can become activated during amyotrophic lateral sclerosis. These cells are also involved in inflammation and appear to accelerate disease progression. We have determined part of the genetic program underlying the activation of inflammatory pathways in microglia. We will attempt to block this genetic program as a way to slow down progression of disease in an animal model of ALS, and then translate this type of therapy to treatment of human ALS.
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Gladstone Institutes, UCSF
Two recently discovered genes that have been associated with both familial and sporadic forms of ALS encode the related proteins TDP43 and FUS cause neuron death in ALS.
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