Therapeutic reduction of ataxin-2 extends lifespan and reduces pathology in TDP-43 mice

Lindsay A. Becker, Brenda Huang, Gregor Bieri, Rosanna Ma, David A. Knowles, Paymaan Jafar-Nejad, James Messing, Hong Joo Kim, Armand Soriano, Georg Auburger, Stefan M. Pulst, J. Paul Taylor, Frank Rigo and Aaron D. Gitler ()
Additional contact information
Lindsay A. Becker: Stanford University School of Medicine
Brenda Huang: Stanford University School of Medicine
Gregor Bieri: Stanford University School of Medicine
Rosanna Ma: Stanford University School of Medicine
David A. Knowles: Stanford University School of Medicine
Paymaan Jafar-Nejad: Ionis Pharmaceuticals
James Messing: St. Jude Children’s Research Hospital
Hong Joo Kim: St. Jude Children’s Research Hospital
Armand Soriano: Ionis Pharmaceuticals
Georg Auburger: Experimental Neurology, Goethe University
Stefan M. Pulst: Experimental Neurology, Goethe University
J. Paul Taylor: St. Jude Children’s Research Hospital
Frank Rigo: Ionis Pharmaceuticals
Aaron D. Gitler: Stanford University School of Medicine

Nature, 2017, vol. 544, issue 7650, 367-371

Abstract: A decrease in ataxin-2 levels leads to a reduction in the aggregation of TDP-43, markedly increased lifespan and improved motor function in a transgenic mouse model of TDP-43 proteinopathy.

Date: 2017
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DOI: 10.1038/nature22038

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