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

Author:

Yu-Wai-Man Patrick1234ORCID,Newman Nancy J.5,Carelli Valerio67ORCID,Moster Mark L.8,Biousse Valerie5,Sadun Alfredo A.9,Klopstock Thomas101112ORCID,Vignal-Clermont Catherine1314ORCID,Sergott Robert C.8ORCID,Rudolph Günther15ORCID,La Morgia Chiara67ORCID,Karanjia Rustum916,Taiel Magali17,Blouin Laure17,Burguière Pierre17ORCID,Smits Gerard18,Chevalier Caroline17,Masonson Harvey18,Salermo Yordak18,Katz Barrett18ORCID,Picaud Serge19ORCID,Calkins David J.20,Sahel José-Alain14192122ORCID

Affiliation:

1. Cambridge Centre for Brain Repair and MRC Mitochondrial Biology Unit, Department of Clinical Neurosciences, University of Cambridge, Cambridge CB2 0PY, UK.

2. Cambridge Eye Unit, Addenbrooke’s Hospital, Cambridge University Hospitals, Cambridge CB2 0QQ, UK.

3. Moorfields Eye Hospital, London EC1V 2PD, UK.

4. UCL Institute of Ophthalmology, University College London, London EC1V 9EL, UK.

5. Departments of Ophthalmology, Neurology and Neurological Surgery, Emory University School of Medicine, Atlanta, GA 30322, USA.

6. IRCCS Istituto delle Scienze Neurologiche di Bologna, UOC Clinica Neurologica, 40139 Bologna, Italy.

7. Unit of Neurology, Department of Biomedical and Neuromotor Sciences (DIBINEM), University of Bologna, 40139 Bologna, Italy.

8. Departments of Neurology and Ophthalmology, Wills Eye Hospital and Thomas Jefferson University, Philadelphia, PA 19107, USA.

9. Doheny Eye Institute and UCLA School of Medicine, Los Angeles, CA 90086, USA.

10. Friedrich Baur Institute at the Department of Neurology, University Hospital, LMU Munich, 80336 Munich, Germany.

11. German Center for Neurodegenerative Diseases (DZNE), 80336 Munich, Germany.

12. Munich Cluster for Systems Neurology (SyNergy), 80336 Munich, Germany.

13. Department of Neuro-Ophthalmology and Emergencies, Rothschild Foundation Hospital, 75019 Paris, France.

14. Centre Hospitalier National d’Ophtalmologie des Quinze Vingts, FOReSIGHT, INSERM-DGOS CIC 1423, 75012 Paris, France.

15. Department of Ophthalmology, University Hospital, LMU Munich, 80336 Munich, Germany.

16. Ottawa Hospital Research Institute and University of Ottawa Eye Institute, Ottawa, Ontario K1H 8L6, Canada.

17. GenSight Biologics, 75012 Paris, France.

18. GenSight Biologics, New York, NY 10016, USA.

19. Sorbonne Université, INSERM, CNRS, Institut de la Vision, 75012 Paris, France.

20. The Vanderbilt Eye Institute, Vanderbilt University Medical Center, Nashville, TN 37232, USA.

21. Fondation Ophtalmologique A. de Rothschild, 25-29 Rue Manin, 75019 Paris, France.

22. Department of Ophthalmology, The University of Pittsburgh School of Medicine, Pittsburgh, PA 15213, USA.

Abstract

At 96 weeks after unilateral intravitreal injection of rAAV2/2- ND4 , vision improved in both eyes in 78% of subjects affected with LHON.

Funder

GenSight Biologics

Publisher

American Association for the Advancement of Science (AAAS)

Subject

General Medicine

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