REFLECT Trial: Efficacy and Safety of Bilateral Gene Therapy for Leber Hereditary Optic Neuropathy

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Identifier 20220215_nanos_sciplatform3_06
Title REFLECT Trial: Efficacy and Safety of Bilateral Gene Therapy for Leber Hereditary Optic Neuropathy
Creator Nancy J. Newman; Patrick Yu-Wai-Man; Valerio Carelli; Prem Subramanian; Mark Moster; An-Guor Wang; Sean Donahue; Bart Leroy; Valérie Biousse; Catherine Vignal-Clermont; Alfredo Sadun; Gema Rebolleda Fernández; Elizabeth Fortin; Rudrani Banik; Laure Blouin; Michel Roux; Magali Taiel; José-Alain Sahe
Affiliation (NJN) (VB) Emory University School of Medicine, Atlanta, GA; (PYWM) Moorfields Eye Hospital, London, United Kingdom of Great Britain and Northern Ireland; (VC) IRCCS Istituto delle Scienze Neurologiche di Bologna, Bologna, Italy; (PS) University of Colorado School of Medicine, Aurora, CO; (MM) Wills Eye Hospital, Philadelphia, PA; (AGW) Taipei Veterans General Hospital, Taipei, Taiwan; (SD) Vanderbilt Eye Institute, Nashville, TN; (BL) Ghent University Hospital, Ghent, Belgium; (CVC) Centre Hospitalier National d'Ophtalmologie des Quinze Vingts, Paris, France; (AS) Doheny Eye Institute, Los Angeles, CA; (GRF) Ramon y Cajal Hospital,Madrid, Spain; (EF) Massachusetts Eye and Ear Infirmary, Boston, MA; (RB) Icahn School of Medicine at Mount Sinai, New York, NY; (LB) (MR) (MT) GenSight Biologics, Paris, France; (JAS) University of Pittsburgh School of Medicine, Pittsburgh, PA
Subject Genetic Disease; Optic Neuropathy; Retina
Description Leber Hereditary Optic Neuropathy (LHON) is a blinding orphan disease leading to bilateral vision loss. Lenadogene nolparvovec (rAAV2/2-ND4) is a gene therapy developed for the treatment of LHON caused by a mutation in the ND4 gene.
Date 2022-02
Language eng
Format application/pdf
Format Creation Microsoft PowerPoint
Type Text
Source 2022 North American Neuro-Ophthalmology Society Annual Meeting
Relation is Part of NANOS Annual Meeting 2022: Scientific Platform Session III
Collection Neuro-Ophthalmology Virtual Education Library: NANOS Annual Meeting Collection: https://novel.utah.edu/collection/nanos-annual-meeting-collection/
Publisher North American Neuro-Ophthalmology Society
Holding Institution Spencer S. Eccles Health Sciences Library, University of Utah
Rights Management Copyright 2022. For further information regarding the rights to this collection, please visit: https://NOVEL.utah.edu/about/copyright
ARK ark:/87278/s6dv7xd9
Setname ehsl_novel_nam
ID 2191987
Reference URL https://collections.lib.utah.edu/ark:/87278/s6dv7xd9
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