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Potential new drug target in Lou Gehrig's disease

November 14, 2011
Rockefeller University Press
Two proteins conspire to promote Lou Gehrig's disease, according to a new study.

Two proteins conspire to promote a lethal neurological disease, according to a study published online this week in the Journal of Experimental Medicine.

Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease, is a devastating neurodegenerative disorder that results in progressive loss of motor function and ultimately death. More than 90% of ALS cases have no known genetic cause or family history. However, in some patients, spinal cord cells contain unusual accumulations of a protein called TDP-43.

Jean-Pierre Julien and colleagues at Laval University in Quebec now find that TDP-43 binds to an inflammatory protein called NF-kB p65 in the spinal cords of ALS patients but not of healthy individuals. TDP-43 and p65 were also more abundant in ALS than healthy spinal cords. In spinal cord cells called microglia, TDP-43 and p65 cooperated to ramp up production of factors capable of promoting inflammation and killing nearby neurons. In a mouse model of ALS, treatment with an agent capable of blocking p65 activity minimized neuron loss and eased disease symptoms.

These findings highlight p65 as a potential therapeutic target for this debilitating disorder.

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The above post is reprinted from materials provided by Rockefeller University Press. Note: Materials may be edited for content and length.

Journal Reference:

  1. Vivek Swarup, Daniel Phaneuf, Nicolas Dupré, Susanne Petri, Michael Strong, Jasna Kriz, and Jean-Pierre Julien. Deregulation of TDP-43 in amyotrophic lateral sclerosis triggers nuclear factor κB–mediated pathogenic pathways. Journal of Experimental Medicine, 2011 DOI: 10.1084/jem.20111313

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Rockefeller University Press. "Potential new drug target in Lou Gehrig's disease." ScienceDaily. ScienceDaily, 14 November 2011. <>.
Rockefeller University Press. (2011, November 14). Potential new drug target in Lou Gehrig's disease. ScienceDaily. Retrieved October 5, 2015 from
Rockefeller University Press. "Potential new drug target in Lou Gehrig's disease." ScienceDaily. (accessed October 5, 2015).

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