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

Author:

Gendron Tania F.12,Chew Jeannie12,Stankowski Jeannette N.1,Hayes Lindsey R.3,Zhang Yong-Jie12ORCID,Prudencio Mercedes12ORCID,Carlomagno Yari1ORCID,Daughrity Lillian M.1,Jansen-West Karen1,Perkerson Emilie A.1,O’Raw Aliesha1ORCID,Cook Casey12,Pregent Luc1ORCID,Belzil Veronique1ORCID,van Blitterswijk Marka12ORCID,Tabassian Lilia J.1,Lee Chris W.12,Yue Mei1,Tong Jimei1,Song Yuping1,Castanedes-Casey Monica1ORCID,Rousseau Linda1ORCID,Phillips Virginia1ORCID,Dickson Dennis W.12,Rademakers Rosa12ORCID,Fryer John D.12,Rush Beth K.4,Pedraza Otto4,Caputo Ana M.5,Desaro Pamela5ORCID,Palmucci Carla5ORCID,Robertson Amelia5ORCID,Heckman Michael G.6,Diehl Nancy N.6,Wiggs Edythe7,Tierney Michael7,Braun Laura7,Farren Jennifer7,Lacomis David8ORCID,Ladha Shafeeq9ORCID,Fournier Christina N.10,McCluskey Leo F.11,Elman Lauren B.11,Toledo Jon B.1213,McBride Jennifer D.13,Tiloca Cinzia14ORCID,Morelli Claudia14,Poletti Barbara14ORCID,Solca Federica14ORCID,Prelle Alessandro15ORCID,Wuu Joanne16,Jockel-Balsarotti Jennifer17ORCID,Rigo Frank18,Ambrose Christine19,Datta Abhishek20,Yang Weixing20ORCID,Raitcheva Denitza21ORCID,Antognetti Giovanna22,McCampbell Alexander23,Van Swieten John C.24,Miller Bruce L.25,Boxer Adam L.25,Brown Robert H.26,Bowser Robert9,Miller Timothy M.17,Trojanowski John Q.13,Grossman Murray11,Berry James D.27,Hu William T.10,Ratti Antonia1428,Traynor Bryan J.29,Disney Matthew D.30,Benatar Michael16ORCID,Silani Vincenzo1428ORCID,Glass Jonathan D.1031,Floeter Mary Kay7,Rothstein Jeffrey D.3,Boylan Kevin B.5,Petrucelli Leonard12ORCID

Affiliation:

1. Department of Neuroscience, Mayo Clinic, Jacksonville, FL 32224, USA.

2. Mayo Graduate School, Mayo Clinic, Jacksonville, FL 32224, USA.

3. Brain Science Institute and Department of Neurology, Johns Hopkins University, Baltimore, MD 21205, USA.

4. Department of Psychiatry and Psychology, Mayo Clinic, Jacksonville, FL 32224, USA.

5. Department of Neurology, Mayo Clinic, Jacksonville, FL 32224, USA.

6. Section of Biostatistics, Mayo Clinic, Jacksonville, FL 32224, USA.

7. Motor Neuron Disorders Unit, National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD 20892, USA.

8. Departments of Neurology and Pathology, University of Pittsburgh School of Medicine and the University of Pittsburgh Medical Center, Pittsburgh, PA 15213, USA.

9. Departments of Neurology and Neurobiology, Barrow Neurological Institute, Phoenix, AZ 85013, USA.

10. Department of Neurology, Emory University School of Medicine, Atlanta, GA 30322, USA.

11. Department of Neurology and the Penn Frontotemporal Degeneration Center, University of Pennsylvania, Philadelphia, PA 19104, USA.

12. Department of Neurology, Houston Methodist Neurological Institute, Houston, TX 77030, USA.

13. Center for Neurodegenerative Disease Research, Department of Pathology and Laboratory Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA.

14. Department of Neurology–Stroke Unit and Laboratory of Neuroscience, IRCCS Istituto Auxologico Italiano, Milan, Italy.

15. Department of Neurology and Stroke Unit, Ospedale Maggiore di Crema, Crema, Italy.

16. Department of Neurology, University of Miami, Miami, FL 33136, USA.

17. Department of Neurology, Washington University School of Medicine, St. Louis, MO 63110, USA.

18. Ionis Pharmaceuticals, 2855 Gazelle Court, Carlsbad, CA 92010, USA.

19. Aleta Biotherapeutics, Natick, MA 01760, USA.

20. Protein Chemistry, Biogen Idec, Cambridge, MA 02142, USA.

21. Global Biomarker and Drug Discovery, Biogen Idec, Cambridge, MA 02142, USA.

22. Biologics Drug Discovery, Biogen Idec, Cambridge, MA 02142, USA.

23. Neurology Research, Biogen Idec, Cambridge, MA 02142, USA.

24. Department of Neurology, Erasmus MC, University Medical Centre, Rotterdam, Netherlands.

25. Department of Neurology, Memory and Aging Center, University of California, San Francisco, San Francisco, CA 94158, USA.

26. Department of Neurology, University of Massachusetts Medical School, Worcester, MA 01655, USA.

27. Neurological Clinical Research Institute, Massachusetts General Hospital, Boston, MA 02114, USA.

28. Department of Pathophysiology and Transplantation, “Dino Ferrari” Centre, Università degli Studi di Milano, Milan, Italy.

29. Neuromuscular Diseases Research Section, Laboratory of Neurogenetics, National Institute of Aging, National Institutes of Health, Bethesda, MD 20892, USA.

30. Department of Chemistry, The Scripps Research Institute, Scripps Florida, Jupiter, FL 33458, USA.

31. Department of Pathology, Emory University School of Medicine, Atlanta, GA 30322, USA.

Abstract

Poly(GP) proteins are a promising pharmacodynamic marker for developing and testing therapeutics for treating C9ORF72 -associated amyotrophic lateral sclerosis.

Funder

U.S. Department of Defense

National Institute on Aging

National Institute of Neurological Disorders and Stroke

Amyotrophic Lateral Sclerosis Association

Muscular Dystrophy Association

ALS Therapy Alliance

Brain Science Institute, Johns Hopkins University

Publisher

American Association for the Advancement of Science (AAAS)

Subject

General Medicine

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