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Bilateral visual improvement with unilateral gene therapy injection for Leber hereditary optic neuropathy

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Science Translational Medicine  09 Dec 2020:
Vol. 12, Issue 573, eaaz7423
DOI: 10.1126/scitranslmed.aaz7423

Article Information

vol. 12 no. 573

Print ISSN: 
Online ISSN: 
  • Received for publication October 5, 2019
  • Resubmitted March 17, 2020
  • Accepted for publication September 2, 2020
  • .

Author Information

  1. Patrick Yu-Wai-Man1,2,3,4,*,
  2. Nancy J. Newman5,
  3. Valerio Carelli6,7,
  4. Mark L. Moster8,
  5. Valerie Biousse5,
  6. Alfredo A. Sadun9,
  7. Thomas Klopstock10,11,12,
  8. Catherine Vignal-Clermont13,14,
  9. Robert C. Sergott8,
  10. Günther Rudolph15,
  11. Chiara La Morgia6,7,
  12. Rustum Karanjia9,16,
  13. Magali Taiel17,
  14. Laure Blouin17,
  15. Pierre Burguière17,
  16. Gerard Smits18,
  17. Caroline Chevalier17,
  18. Harvey Masonson18,
  19. Yordak Salermo18,
  20. Barrett Katz18,
  21. Serge Picaud19,
  22. David J. Calkins20 and
  23. José-Alain Sahel14,19,21,22,*
  1. 1Cambridge Centre for Brain Repair and MRC Mitochondrial Biology Unit, Department of Clinical Neurosciences, University of Cambridge, Cambridge CB2 0PY, UK.
  2. 2Cambridge Eye Unit, Addenbrooke’s Hospital, Cambridge University Hospitals, Cambridge CB2 0QQ, UK.
  3. 3Moorfields Eye Hospital, London EC1V 2PD, UK.
  4. 4UCL Institute of Ophthalmology, University College London, London EC1V 9EL, UK.
  5. 5Departments of Ophthalmology, Neurology and Neurological Surgery, Emory University School of Medicine, Atlanta, GA 30322, USA.
  6. 6IRCCS Istituto delle Scienze Neurologiche di Bologna, UOC Clinica Neurologica, 40139 Bologna, Italy.
  7. 7Unit of Neurology, Department of Biomedical and Neuromotor Sciences (DIBINEM), University of Bologna, 40139 Bologna, Italy.
  8. 8Departments of Neurology and Ophthalmology, Wills Eye Hospital and Thomas Jefferson University, Philadelphia, PA 19107, USA.
  9. 9Doheny Eye Institute and UCLA School of Medicine, Los Angeles, CA 90086, USA.
  10. 10Friedrich Baur Institute at the Department of Neurology, University Hospital, LMU Munich, 80336 Munich, Germany.
  11. 11German Center for Neurodegenerative Diseases (DZNE), 80336 Munich, Germany.
  12. 12Munich Cluster for Systems Neurology (SyNergy), 80336 Munich, Germany.
  13. 13Department of Neuro-Ophthalmology and Emergencies, Rothschild Foundation Hospital, 75019 Paris, France.
  14. 14Centre Hospitalier National d’Ophtalmologie des Quinze Vingts, FOReSIGHT, INSERM-DGOS CIC 1423, 75012 Paris, France.
  15. 15Department of Ophthalmology, University Hospital, LMU Munich, 80336 Munich, Germany.
  16. 16Ottawa Hospital Research Institute and University of Ottawa Eye Institute, Ottawa, Ontario K1H 8L6, Canada.
  17. 17GenSight Biologics, 75012 Paris, France.
  18. 18GenSight Biologics, New York, NY 10016, USA.
  19. 19Sorbonne Université, INSERM, CNRS, Institut de la Vision, 75012 Paris, France.
  20. 20The Vanderbilt Eye Institute, Vanderbilt University Medical Center, Nashville, TN 37232, USA.
  21. 21Fondation Ophtalmologique A. de Rothschild, 25-29 Rue Manin, 75019 Paris, France.
  22. 22Department of Ophthalmology, The University of Pittsburgh School of Medicine, Pittsburgh, PA 15213, USA.
  1. *Corresponding author. Email: sahelja{at} (J.-A.S.); py237{at} (P.Y.-W.-M.)


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Article usage: December 2020 to June 2021

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Jan 20212834502256
Feb 2021125310776
Mar 20211244101107
Apr 20218519266
May 202189171850
Jun 202136084929

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