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Newman NJ, Yu-Wai-Man P, Carelli V, et al. Intravitreal Gene Therapy vs. Natural History in Patients With Leber Hereditary Optic Neuropathy Carrying the m.11778G>A . Front Neurol. 2021;12:662838doi: 10.3389/fneur.2021.662838.
Newman, N. J., Yu-Wai-Man, P., Carelli, V., Biousse, V., Moster, M. L., Vignal-Clermont, C., Sergott, R. C., Klopstock, T., Sadun, A. A., Girmens, J. F., La Morgia, C., DeBusk, A. A., Jurkute, N., Priglinger, C., Karanjia, R., Josse, C., Salzmann, J., Montestruc, F., Roux, M., Taiel, M., & Sahel, J. A. (2021). Intravitreal Gene Therapy vs. Natural History in Patients With Leber Hereditary Optic Neuropathy Carrying the m.11778G>A . Frontiers in neurology, 12662838. https://doi.org/10.3389/fneur.2021.662838
Newman, Nancy J, et al. "Intravitreal Gene Therapy vs. Natural History in Patients With Leber Hereditary Optic Neuropathy Carrying the m.11778G>A ." Frontiers in neurology vol. 12 (2021): 662838. doi: https://doi.org/10.3389/fneur.2021.662838
Newman NJ, Yu-Wai-Man P, Carelli V, Biousse V, Moster ML, Vignal-Clermont C, Sergott RC, Klopstock T, Sadun AA, Girmens JF, La Morgia C, DeBusk AA, Jurkute N, Priglinger C, Karanjia R, Josse C, Salzmann J, Montestruc F, Roux M, Taiel M, Sahel JA. Intravitreal Gene Therapy vs. Natural History in Patients With Leber Hereditary Optic Neuropathy Carrying the m.11778G>A . Front Neurol. 2021 May 24;12:662838. doi: 10.3389/fneur.2021.662838. eCollection 2021. PMID: 34108929; PMCID: PMC8181419.
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Front Neurol. 2021 May 24;12:662838. doi: 10.3389/fneur.2021.662838. eCollection 2021.

Intravitreal Gene Therapy vs. Natural History in Patients With Leber Hereditary Optic Neuropathy Carrying the m.11778G>A .

Frontiers in neurology

Nancy J Newman, Patrick Yu-Wai-Man, Valerio Carelli, Valerie Biousse, Mark L Moster, Catherine Vignal-Clermont, Robert C Sergott, Thomas Klopstock, Alfredo A Sadun, Jean-François Girmens, Chiara La Morgia, Adam A DeBusk, Neringa Jurkute, Claudia Priglinger, Rustum Karanjia, Constant Josse, Julie Salzmann, François Montestruc, Michel Roux, Magali Taiel, José-Alain Sahel

Affiliations

  1. Departments of Ophthalmology, Neurology and Neurological Surgery, Emory University School of Medicine, Atlanta, GA, United States.
  2. Cambridge Centre for Brain Repair and MRC Mitochondrial Biology Unit, Department of Clinical Neurosciences, University of Cambridge, Cambridge, United Kingdom.
  3. Cambridge Eye Unit, Addenbrooke's Hospital, Cambridge University Hospitals, Cambridge, United Kingdom.
  4. Moorfields Eye Hospital National Health Service Foundation Trust, London, United Kingdom.
  5. UCL Institute of Ophthalmology, University College London, London, United Kingdom.
  6. Istituto di Ricovero e Cura a Carattere Scientifico Istituto delle Scienze Neurologiche di Bologna, Unitá Operativa Compless Clinica Neurologica, Bologna, Italy.
  7. Unit of Neurology, Department of Biomedical and Neuromotor Sciences, University of Bologna, Bologna, Italy.
  8. Departments of Neurology and Ophthalmology, Wills Eye Hospital and Thomas Jefferson University, Philadelphia, PA, United States.
  9. Department of Neuro Ophthalmology and Emergencies, A. de Rothschild Foundation Hospital, Paris, France.
  10. Centre d'investigation Clinique, Centre Hospitalier National d'Ophtalmologie des Quinze Vingts, Paris, France.
  11. Department of Neurology, Friedrich-Baur-Institute, University Hospital, Ludwig-Maximilians-University Munich, Munich, Germany.
  12. German Center for Neurodegenerative Diseases, Munich, Germany.
  13. Munich Cluster for Systems Neurology, Munich, Germany.
  14. Doheny Eye Institute, School of Medicine, University of California, Los Angeles, Los Angeles, CA, United States.
  15. Department of Ophthalmology, University Hospital, Ludwig-Maximilians-University Munich, Munich, Germany.
  16. Department of Ophthalmology, University of Ottawa Eye, Ottawa, ON, Canada.
  17. eXYSTAT, Data Management and Statistic, Malakoff, France.
  18. Medical and Regulatory Consulting, Paris, France.
  19. GenSight Biologics, Paris, France.
  20. Sorbonne Université, INSERM, CNRS, Institut de la Vision, Paris, France.
  21. A. de Rothschild Foundation Hospital, Paris, France.
  22. Department of Ophthalmology, The University of Pittsburgh School of Medicine, Pittsburgh, PA, United States.
  23. CHNO des Quinze-Vingts, Institut Hospitalo-Universitaire FOReSIGHT, INSERM-DGOS CIC 1423, Paris, France.

PMID: 34108929 PMCID: PMC8181419 DOI: 10.3389/fneur.2021.662838

[No abstract available.]

Copyright © 2021 Newman, Yu-Wai-Man, Carelli, Biousse, Moster, Vignal-Clermont, Sergott, Klopstock, Sadun, Girmens, La Morgia, DeBusk, Jurkute, Priglinger, Karanjia, Josse, Salzmann, Montestruc, Roux, Taiel and Sahel.

Keywords: Leber hereditary optic neuropathy; ND4; gene therapy; natural history; visual acuity

Conflict of interest statement

NN is a consultant for GenSight, Santhera Pharmaceuticals, and Stealth BioTherapeutics, has received research support from GenSight and Santhera Pharmaceuticals, has served on the Data Safety Monitori

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