Assessment of rAAV2/2-ND4 Gene Therapy Efficacy in LHON Using an External Control Group
| Identifier | 20210222_nanos_sciplatform2_03-slides |
| 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 | application/pdf |
| Format Creation | Microsoft PowerPoint |
| Type | Text |
| 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: 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 2021. For further information regarding the rights to this collection, please visit: https://NOVEL.utah.edu/about/copyright |
| ARK | ark:/87278/s6xx0hmg |
| Setname | ehsl_novel_nam |
| ID | 1694138 |
| Reference URL | https://collections.lib.utah.edu/ark:/87278/s6xx0hmg |