Brain’s Immune System is Key to Recovery from Motor Neuron Degeneration in ALS Animal Model

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Results in Penn study point to new approaches for ALS therapy

 

The selective demise of motor neurons is the hallmark of Lou Gehrig’s disease, also known as amyotrophic lateral sclerosis (ALS). Yet neurologists have suspected there are other types of brain cells involved in the progression of this disorder — perhaps protection from it, which could light the way to treatment methods for the incurable disease. To get to the bottom of this question, researchers in the Perelman School of Medicine at the University of Pennsylvania engineered mice in which the damage caused by a mutant human TDP-43 protein could be reversed by one type of brain immune cell. TDP-43 is a protein that misfolds and accumulates in the motor areas of the brains of ALS patients. 

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They found that microglia, the first and primary immune response cells in the brain and spinal cord, are essential for dealing with TDP-43-associated neuron death. This study is the first to demonstrate how healthy microglia respond to pathological TDP-43 in a living animal.

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