Assessment of rAAV2/2-ND4 Gene Therapy Efficacy in LHON Using an External Control Group

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Identifier 20210222_nanos_sciplatform2_03-video
Title Assessment of rAAV2/2-ND4 Gene Therapy Efficacy in LHON Using an External Control Group
Creator Nancy J. Newman; Patrick Yu-Wai-Man; Valerio Carelli; Mark Moster; Catherine Vignal-Clermont; Alfredo Sadun; Chiara La Morgia; Francesco Bandello; Lorena Castillo Campillo; Stéphanie Leruez; Dean Cestari; Rod Foroozan; Rustum Karanija; Laure Blouin; Michel Roux; Magali Taiel; José-Alain Sahel
Affiliation (NJN) Emory University School of Medicine, Atlanta, GA; (PYWM) Cambridge Centre for Brain Repair and MRC Mitochondrial Biology Unit, Cambridge, London, United Kingdom of Great Britain and Northern Ireland; (VC) (CLM) Unit of Neurology, Department of Biomedical and Neuromotor Sciences, Bologna, Italy; (MM) Wills Eye Hospital and Thomas Jefferson University, Philadelphia, PA; (CVC) Centre Hospitalier National d'Ophtalmologie des Quinze Vingts, Paris, France; (AS) Doheny Eye Institute/ UCLA School of Medicine, Los Angeles, CA; (FB) Department of Ophthalmology Vita-Salute San Raffaele University, Milan, Italy; (LCC) Institut Catala de Retina, Barcelona, Spain; (SL) CHU Angers - Hopital Hotel Dieu, Angers, France; (DC) Massachusetts Eye and Ear Infirmary, Boston, MA; (RF) Alkek Eye Center, Houston, TX; (RK) Doheny Eye Institute / UCLA School of Medicine, Los Angeles, CA; (LB) (MR) (MT) GenSight Biologics, Paris, France; (JAS) Institut de la Vision, Paris, France; Pittsburgh School of Medicine, Pittsburgh, PA
Subject Optic Neuropathy; Genetic Disease
Description The two Phase-3 multi-center clinical trials RESCUE and REVERSE showed sustained bilateral improvement of best-corrected visual acuity (BCVA) following unilateral injection of rAAV2/2-ND4 gene therapy for Leber Hereditary Optic Neuropathy (LHON). This bilateral treatment effect was compared to an external control group comprised of untreated ND4-LHON patients.
Date 2021-02
Language eng
Format video/mp4
Type Image/MovingImage
Source 2021 North American Neuro-Ophthalmology Society Annual Meeting
Relation is Part of NANOS Annual Meeting 2021: Scientific Platform Session II
Collection Neuro-ophthalmology Virtual Education Library: NOVEL http://NOVEL.utah.edu
Publisher Spencer S. Eccles Health Sciences Library, University of Utah
Holding Institution North American Neuro-Ophthalmology Association. NANOS Executive Office 5841 Cedar Lake Road, Suite 204, Minneapolis, MN 55416
Rights Management Copyright 2021. For further information regarding the rights to this collection, please visit: https://NOVEL.utah.edu/about/copyright
ARK ark:/87278/s6xh5qck
Context URL The NANOS Annual Meeting Neuro-Ophthalmology Collection: https://novel.utah.edu/collection/NAM/toc/
Setname ehsl_novel_nam
ID 1697222
Reference URL https://collections.lib.utah.edu/ark:/87278/s6xh5qck
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