Indirect Comparison of Lenadogene Nolparvovec Gene Therapy Versus Natural History in Patients with Leber Hereditary Optic Neuropathy Carrying the m.11778G>A MT-ND4 Mutation
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Published:2022-11-30
Issue:1
Volume:12
Page:401-429
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ISSN:2193-8245
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Container-title:Ophthalmology and Therapy
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language:en
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Short-container-title:Ophthalmol Ther
Author:
Carelli ValerioORCID, Newman Nancy J., Yu-Wai-Man Patrick, Biousse Valerie, Moster Mark L., Subramanian Prem S., Vignal-Clermont Catherine, Wang An-Guor, Donahue Sean P., Leroy Bart P., Sergott Robert C., Klopstock Thomas, Sadun Alfredo A., Rebolleda Fernández Gema, Chwalisz Bart K., Banik Rudrani, Girmens Jean François, La Morgia Chiara, DeBusk Adam A., Jurkute Neringa, Priglinger Claudia, Karanjia Rustum, Josse Constant, Salzmann Julie, Montestruc François, Roux Michel, Taiel Magali, Sahel José-Alain, Carelli Valerio, Barboni Piero, Carbonelli Michele, Di Vito Lidia, Amore Giulia, Contin Manuela, Mohamed Susan, La Morgia Chiara, Silvestri Sara, D’Agati Pietro, Newman Nancy J, Biousse Valérie, Hubbard George Baker, O’Keefe Ghazala, Hendrick Andrew M., Dattilo Michael, Peragallo Jason H., Hawy Eman, DuBois Lindreth, Gibbs Deborah, Filho Alcides Fernandes, Dobbs Jannah, Yu-Wai-Man Patrick, Acheson James, Boston Hayley, Eleftheriadou Maria, Esposti Simona, Gemenetzi Maria, Leitch-Devlin Lauren, Tucker William R., Jurkute Neringa, Burale Asma, Anand Shweta, Memon Muhammad A., Hussain Rima, Jorany Rasha, Sheel Priyansha, Moster Mark L., Sergott Robert C., SantaMaria Melissa, Tollis Heather, DeBusk Adam A, Haller Julia A., Massini Maria, Subramanian Prem S., Pecen Paula, Mathias Marc, Preston Mary, Cho Steve, Sahel José A., Vignal-Clermont Catherine, Girmens Jean François, Hage Rabih, Plaine Lise, Khemliche Wahiba, Wang An-Guor, Cheng Hui-Chen, Chen Celia, Hwang Jeong-Min, Sun Chuanbin, Donahue Sean, Patel Shriji, Gangaputra Sapna, Barrett Megan, Ruark Scott, Wilkins Saige, Leroy Bart P., De Zaeytijd Julie, Van Cauwenbergh Caroline, Verhauwen Hilde, Klopstock Thomas, Catarino Claudia B., Priglinger Claudia, Priglinger Siegfried, Rudolph Günther, von Livonius Bettina Stephan Thurau, Muth Daniel, Wolf Armin, Al-Tamami Jasmina, Pressler Angelika, Schertler Cosima, Hildebrandt Martin, Neuenhahn Michael, Sadun Alfredo A., Heilweil Gad, Karanjia Rustum, Tsui Irena, Rebolleda Fernández Gema, Urquijo Laia Jaumendreu, Negrete Muñoz Francisco J., Fortin Elizabeth, Chwalisz Bart K., Cestari Dean, Banik Rudrani, Tai Katy, Castillo Lorena, Garcia Virginia, Bandello Antonio Morilla Francesco, Barboni Piero, Cascavilla Maria Lucia, Battista Marco, Calcagno Francesca, Pina Adelaide, Leruez Stéphanie, Forooza Rod,
Funder
GenSight Biologics Italian Ministry of Health Italian Ministry of University and Research Telethon-Italy patients’ organizations MITOCON and IFOND, and patients’ donations NIH/NEI NIH/NINDS UK National Institute of Health Research UK Medical Research Council Fight for Sight UK Isaac Newton Trust Moorfields Eye Charity Addenbrooke’s Charitable Trust National Eye Research Centre International Foundation for Optic Nerve Disease NIHR as part of the Rare Diseases Translational Research Collaboration NIHR Cambridge Biomedical Research Centre National Institute for Health Research Biomedical Research Centre at Moorfields Eye Hospital NHS Foundation Trust and UCL Institute of Ophthalmology Research Foundation - Flanders, Belgium Concerted Research Action of the Special Research Fund Ghent University German Network for Mitochondrial Disorders E-Rare project GENOMIT Agence Nationale de la Recherche, Programme Investissements d'Avenir LabEx LIFESENSES
Publisher
Springer Science and Business Media LLC
Reference59 articles.
1. Yu-Wai-Man P, Votruba M, Burté F, La Morgia C, Barboni P, Carelli V. A neurodegenerative perspective on mitochondrial optic neuropathies. Acta Neuropathol. 2016;132:789–806. 2. Chen BS, Holzinger E, Taiel M, Yu-Wai-Man P. The impact of Leber hereditary optic neuropathy on the quality of life of patients and their relatives: a qualitative study. J Neuro-Ophthalmol. 2022;42:316–22. 3. Riordan-Eva P, Sanders MD, Govan GG, Sweeney MG, Da CJ, Harding AE. The clinical features of Leber’s hereditary optic neuropathy defined by the presence of a pathogenic mitochondrial DNA mutation. Brain. 1995;118:319–37. 4. Newman NJ, Lott MT, Wallace DC. The clinical characteristics of pedigrees of Leber’s hereditary optic neuropathy with the 11778 mutation. Am J Ophthalmol. 1991;111:750–62. 5. Yu-Wai-Man P, Turnbull DM, Chinnery PF. Leber hereditary optic neuropathy. J Med Genet. 2002;39:162–9.
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