Research ArticleParkinson’s Disease

A patient-based model of RNA mis-splicing uncovers treatment targets in Parkinson’s disease

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Science Translational Medicine  09 Sep 2020:
Vol. 12, Issue 560, eaau3960
DOI: 10.1126/scitranslmed.aau3960

Article Information

vol. 12 no. 560

PubMed: 
Print ISSN: 
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History: 
  • Received for publication June 6, 2018
  • Resubmitted January 24, 2020
  • Accepted for publication June 22, 2020
  • .

Author Information

  1. Ibrahim Boussaad1,*,
  2. Carolin D. Obermaier1,2,*,
  3. Zoé Hanss1,
  4. Dheeraj R. Bobbili1,
  5. Silvia Bolognin1,
  6. Enrico Glaab1,
  7. Katarzyna Wołyńska3,
  8. Nicole Weisschuh4,
  9. Laura De Conti5,
  10. Caroline May6,
  11. Florian Giesert7,8,9,
  12. Dajana Grossmann1,
  13. Annika Lambert1,
  14. Susanne Kirchen1,
  15. Maria Biryukov1,
  16. Lena F. Burbulla10,
  17. Francois Massart1,
  18. Jill Bohler1,
  19. Gérald Cruciani1,
  20. Benjamin Schmid2,
  21. Annerose Kurz-Drexler7,
  22. Patrick May1,
  23. Stefano Duga11,12,
  24. Christine Klein13,
  25. Jens C. Schwamborn1,
  26. Katrin Marcus6,
  27. Dirk Woitalla14,
  28. Daniela M. Vogt Weisenhorn7,9,
  29. Wolfgang Wurst7,8,9,15,
  30. Marco Baralle5,
  31. Dimitri Krainc10,
  32. Thomas Gasser2,16,
  33. Bernd Wissinger4 and
  34. Rejko Krüger1,2,17,18,
  1. 1LCSB, Luxembourg Centre for Systems Biomedicine, University of Luxembourg, 4362 Esch-Sur-Alzette, Luxembourg.
  2. 2Department of Neurodegenerative Diseases and Hertie-Institute for Clinical Brain Research, University of Tübingen, 72076 Tübingen, Germany.
  3. 3Department of Medical Genetics, Poznan University of Medical Sciences, 60-806 Poznan, Poland.
  4. 4Molecular Genetics Laboratory, Institute for Ophthalmic Research, University Clinics Tübingen, 72076 Tübingen, Germany.
  5. 5ICGEB—International Centre for Genetic Engineering and Biotechnology, Padriciano 99, 34149 Trieste, Italy.
  6. 6Medizinisches Proteom-Center, Ruhr-University Bochum, 44801 Bochum, Germany.
  7. 7Helmholtz Zentrum München, Ingolstaedter Landstr. 1, 85764 Neuherberg, Germany.
  8. 8German Center for Neurodegenerative Diseases (DZNE), Site Munich, Feodor-Lynen-Str. 17, 81377 Munich, Germany.
  9. 9Technische Universität München-Weihenstephan, Developmental Genetics, c/o Helmholtz Zentrum München, Ingolstädter Landstr. 1, 85764 Neuherberg, Germany.
  10. 10Department of Neurology, Northwestern University Feinberg School of Medicine, Chicago, IL 60611, USA.
  11. 11Department of Biomedical Sciences, Humanitas University, Via Manzoni 113, 20089 Rozzano, Milan, Italy.
  12. 12Humanitas Clinical and Research center, IRCCS, Via Manzoni 56, 20089 Rozzano, Milan, Italy.
  13. 13Institute of Neurogenetics, University of Luebeck, 23562 Luebeck, Germany.
  14. 14Department of Neurology, St. Josef-Hospital, Ruhr-University Bochum, 44791 Bochum, Germany.
  15. 15Munich Cluster for Systems Neurology (SyNergy), Feodor-Lynen-Str. 17, 81377 Munich, Germany.
  16. 16German Center for Neurodegenerative Diseases (DZNE), 72076 Tübingen, Germany.
  17. 17Department of Neurology and Parkinson Research Clinic, Centre Hospitalier de Luxembourg (CHL), 1210 Luxembourg, Luxembourg.
  18. 18Transversal Translational Medicine, Luxembourg Institute of Health (LIH), 1445 Strassen, Luxembourg.
  1. Corresponding author. Email: rejko.krueger{at}uni.lu
  • * These authors contributed equally to this work.

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