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Biogen Idec And Academic Collaboration: Discovery Of New ALS Gene

February 25, 2015

On February 19th, using advanced DNA sequencing methods, Biogen Idec announces that an international consortium, that includes clinicians and scientists from the company, Columbia University Medical Center (CUMC), and HudsonAlpha Institute for Biotechnology (Huntsville, Alabama) identify a new gene associated with sporadic Amyotrophic Lateral Sclerosis (ALS). The newly associated gene is called TBK1 (TANK-Binding Kinase1). It is also found that a gene, called OPTN, previously thought to play a minor role in ALS, may actually be a major player in the disease.

The study is published in the online edition of the journal Science with the following Abstract:

Amyotrophic lateral sclerosis (ALS) is a devastating neurological disease with no effective treatment. Here we report the results of a moderate-scale sequencing study aimed at identifying new genes contributing to predisposition for ALS. We performed whole exome sequencing of 2,874 ALS patients and compared them to 6,405 controls. Several known ALS genes were found to be associated, and the non-canonical IκB kinase family TANK-Binding Kinase 1 (TBK1) was identified as an ALS gene. TBK1 is known to bind to and phosphorylate a number of proteins involved in innate immunity and autophagy, including optineurin (OPTN) and p62 (SQSTM1/sequestosome), both of which have also been implicated in ALS. These observations reveal a key role of the autophagic pathway in ALS and suggest specific targets for therapeutic intervention.

The discovery of the new gene TBK1 for ALS, is important as it aids in identification of key biological pathways relevant to ALS that now can become the focus of targeted drug development. Several compounds that affect TBK1 signaling have already been developed for use in cancer, where the gene is thought to play a role in tumor cell survival.

Please Note: “Human Chromosomes” Courtesy of Jane Ades, NHGRI.

Copyright © 2012-2015, Orphan Druganaut Blog. All rights reserved.

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