rna-binding proteins in amyotrophic lateral sclerosis and neurodegenerationrna结合蛋白在肌萎缩性脊髓侧索硬化症和神经退化.pdfVIP

Hindawi Publishing Corporation Neurology Research International Volume 2012, Article ID 432780, 5 pages doi:10.1155/2012/432780 Review Article RNA-Binding Proteins in Amyotrophic Lateral Sclerosis and Neurodegeneration Scott E. Ugras1, 2 and James Shorter1, 2 1 Biochemistry and Molecular Biophysics Graduate Group, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA 19104, USA 2 Department of Biochemistry and Biophysics, Perelman School of Medicine, University of Pennsylvania, 805b Stellar-Chance Laboratories, 422 Curie Boulevard, Philadelphia, PA 19104, USA Correspondence should be addressed to James Shorter, jshorter@ Received 26 January 2012; Accepted 26 February 2012 Academic Editor: Jeff Bronstein Copyright © 2012 S. E. Ugras and J. Shorter. This is an open access article distributed under the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. Amyotrophic Lateral Sclerosis (ALS) is an adult onset neurodegenerative disease, which is universally fatal. While the causes of this devastating disease are poorly understood, recent advances have implicated RNA-binding proteins (RBPs) that contain predicted prion domains as a major culprit. Specifically, mutations in the RBPs TDP-43 and FUS can cause ALS. Cytoplasmic mislocalization and inclusion formation are common pathological features of TDP-43 and FUS proteinopathies. Though these RBPs share striking pathological and structural similarities, considerable evidence suggests that the ALS-linked mutations in TDP-43 and FUS can cause disease by disparate mechanisms. In a recent study, Couthouis et al. screened for protein candidates that were also involved in RNA processi