Research ArticleAmyotrophic Lateral Sclerosis

Poly(GP) proteins are a useful pharmacodynamic marker for C9ORF72-associated amyotrophic lateral sclerosis

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Science Translational Medicine  29 Mar 2017:
Vol. 9, Issue 383, eaai7866
DOI: 10.1126/scitranslmed.aai7866

Article Information

vol. 9 no. 383

PubMed: 
Print ISSN: 
Online ISSN: 
History: 
  • Received for publication August 26, 2016
  • Accepted for publication January 13, 2017
  • .

Author Information

  1. Tania F. Gendron1,2,*,
  2. Jeannie Chew1,2,*,
  3. Jeannette N. Stankowski1,*,
  4. Lindsey R. Hayes3,*,
  5. Yong-Jie Zhang1,2,
  6. Mercedes Prudencio1,2,
  7. Yari Carlomagno1,
  8. Lillian M. Daughrity1,
  9. Karen Jansen-West1,
  10. Emilie A. Perkerson1,
  11. Aliesha O’Raw1,
  12. Casey Cook1,2,
  13. Luc Pregent1,
  14. Veronique Belzil1,
  15. Marka van Blitterswijk1,2,
  16. Lilia J. Tabassian1,
  17. Chris W. Lee1,2,
  18. Mei Yue1,
  19. Jimei Tong1,
  20. Yuping Song1,
  21. Monica Castanedes-Casey1,
  22. Linda Rousseau1,
  23. Virginia Phillips1,
  24. Dennis W. Dickson1,2,
  25. Rosa Rademakers1,2,
  26. John D. Fryer1,2,
  27. Beth K. Rush4,
  28. Otto Pedraza4,
  29. Ana M. Caputo5,
  30. Pamela Desaro5,
  31. Carla Palmucci5,
  32. Amelia Robertson5,
  33. Michael G. Heckman6,
  34. Nancy N. Diehl6,
  35. Edythe Wiggs7,
  36. Michael Tierney7,
  37. Laura Braun7,
  38. Jennifer Farren7,
  39. David Lacomis8,
  40. Shafeeq Ladha9,
  41. Christina N. Fournier10,
  42. Leo F. McCluskey11,
  43. Lauren B. Elman11,
  44. Jon B. Toledo12,13,
  45. Jennifer D. McBride13,
  46. Cinzia Tiloca14,
  47. Claudia Morelli14,
  48. Barbara Poletti14,
  49. Federica Solca14,
  50. Alessandro Prelle15,
  51. Joanne Wuu16,
  52. Jennifer Jockel-Balsarotti17,
  53. Frank Rigo18,
  54. Christine Ambrose19,
  55. Abhishek Datta20,
  56. Weixing Yang20,
  57. Denitza Raitcheva21,
  58. Giovanna Antognetti22,
  59. Alexander McCampbell23,
  60. John C. Van Swieten24,
  61. Bruce L. Miller25,
  62. Adam L. Boxer25,
  63. Robert H. Brown26,
  64. Robert Bowser9,
  65. Timothy M. Miller17,
  66. John Q. Trojanowski13,
  67. Murray Grossman11,
  68. James D. Berry27,
  69. William T. Hu10,
  70. Antonia Ratti14,28,
  71. Bryan J. Traynor29,
  72. Matthew D. Disney30,
  73. Michael Benatar16,
  74. Vincenzo Silani14,28,
  75. Jonathan D. Glass10,31,
  76. Mary Kay Floeter7,
  77. Jeffrey D. Rothstein3,
  78. Kevin B. Boylan5 and
  79. Leonard Petrucelli1,2,
  1. 1Department of Neuroscience, Mayo Clinic, Jacksonville, FL 32224, USA.
  2. 2Mayo Graduate School, Mayo Clinic, Jacksonville, FL 32224, USA.
  3. 3Brain Science Institute and Department of Neurology, Johns Hopkins University, Baltimore, MD 21205, USA.
  4. 4Department of Psychiatry and Psychology, Mayo Clinic, Jacksonville, FL 32224, USA.
  5. 5Department of Neurology, Mayo Clinic, Jacksonville, FL 32224, USA.
  6. 6Section of Biostatistics, Mayo Clinic, Jacksonville, FL 32224, USA.
  7. 7Motor Neuron Disorders Unit, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD 20892, USA.
  8. 8Departments of Neurology and Pathology, University of Pittsburgh School of Medicine and the University of Pittsburgh Medical Center, Pittsburgh, PA 15213, USA.
  9. 9Departments of Neurology and Neurobiology, Barrow Neurological Institute, Phoenix, AZ 85013, USA.
  10. 10Department of Neurology, Emory University School of Medicine, Atlanta, GA 30322, USA.
  11. 11Department of Neurology and the Penn Frontotemporal Degeneration Center, University of Pennsylvania, Philadelphia, PA 19104, USA.
  12. 12Department of Neurology, Houston Methodist Neurological Institute, Houston, TX 77030, USA.
  13. 13Center for Neurodegenerative Disease Research, Department of Pathology and Laboratory Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA.
  14. 14Department of Neurology–Stroke Unit and Laboratory of Neuroscience, IRCCS Istituto Auxologico Italiano, Milan, Italy.
  15. 15Department of Neurology and Stroke Unit, Ospedale Maggiore di Crema, Crema, Italy.
  16. 16Department of Neurology, University of Miami, Miami, FL 33136, USA.
  17. 17Department of Neurology, Washington University School of Medicine, St. Louis, MO 63110, USA.
  18. 18Ionis Pharmaceuticals, 2855 Gazelle Court, Carlsbad, CA 92010, USA.
  19. 19Aleta Biotherapeutics, Natick, MA 01760, USA.
  20. 20Protein Chemistry, Biogen Idec, Cambridge, MA 02142, USA.
  21. 21Global Biomarker and Drug Discovery, Biogen Idec, Cambridge, MA 02142, USA.
  22. 22Biologics Drug Discovery, Biogen Idec, Cambridge, MA 02142, USA.
  23. 23Neurology Research, Biogen Idec, Cambridge, MA 02142, USA.
  24. 24Department of Neurology, Erasmus MC, University Medical Centre, Rotterdam, Netherlands.
  25. 25Department of Neurology, Memory and Aging Center, University of California, San Francisco, San Francisco, CA 94158, USA.
  26. 26Department of Neurology, University of Massachusetts Medical School, Worcester, MA 01655, USA.
  27. 27Neurological Clinical Research Institute, Massachusetts General Hospital, Boston, MA 02114, USA.
  28. 28Department of Pathophysiology and Transplantation, “Dino Ferrari” Centre, Università degli Studi di Milano, Milan, Italy.
  29. 29Neuromuscular Diseases Research Section, Laboratory of Neurogenetics, National Institute of Aging, National Institutes of Health, Bethesda, MD 20892, USA.
  30. 30Department of Chemistry, The Scripps Research Institute, Scripps Florida, Jupiter, FL 33458, USA.
  31. 31Department of Pathology, Emory University School of Medicine, Atlanta, GA 30322, USA.
  1. Corresponding author. Email: petrucelli.leonard{at}mayo.edu
    • * These authors contributed equally to this work.

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