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Potential new drug target in Lou Gehrig's disease

Date:
November 14, 2011
Source:
Rockefeller University Press
Summary:
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.

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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.


Story Source:

The above story is based on 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

Cite This Page:

Rockefeller University Press. "Potential new drug target in Lou Gehrig's disease." ScienceDaily. ScienceDaily, 14 November 2011. <www.sciencedaily.com/releases/2011/11/111114133630.htm>.
Rockefeller University Press. (2011, November 14). Potential new drug target in Lou Gehrig's disease. ScienceDaily. Retrieved March 4, 2015 from www.sciencedaily.com/releases/2011/11/111114133630.htm
Rockefeller University Press. "Potential new drug target in Lou Gehrig's disease." ScienceDaily. www.sciencedaily.com/releases/2011/11/111114133630.htm (accessed March 4, 2015).

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