Identifier |
20230314_nanos_sciplatform3_02 |
Title |
Histopathological and Molecular Characterization in Ocular Post-Mortem Analyses Following AAV2 Gene Therapy for LHON |
Creator |
Alfredo Sadun; Leonardo Caporali; Fred Ross-Cisneros; Elisa Boschetti; Nancy Newman; Valerie Biousse; Lindreth DuBois; Henry Liu; Philippe Ancian; Magali Taiel; Valerio Carelli |
Affiliation |
(AS) Doheny Eye Institute, UCLA School of Medicine; (LC) (VC) IRCCS Istituto delle Scienze Neurologiche di Bologna; (FR) Doheny Eye Institute; (EB) University of Bologna; (NN) Departments of Ophthalmology, Neurology, and Neurological Surgery, Emory University School of Medicine, Atlanta, GA; (VB) Departments of Ophthalmology and Neurology, Emory University School of Medicine, Atlanta, GA; (LD) Emory University, Atlanta, GA' (HL) Doheny Eye Center-UCLA; (PA) Charles River; (MT) GenSight Biologics |
Subject |
Optic Neuropathy; Genetic Disease |
Description |
Lenadogene nolparvovec (AAV2-ND4) is a novel AAV2 gene therapy for patients with Leber hereditary optic neuropathy (LHON) carrying the m.11778G>A ND4 mutation. As demonstrated by the RESTORE five-year follow-up study of RESCUE and REVERSE, and the recent therapy/therapy versus therapy/placebo bilateral-injection REFLECT study, unilateral injection of lenadogene nolparvovec induces sustained bilateral improvement in visual acuity more than the reported natural history of this disorder. Non-human-primate studies have documented AAV2-ND4 in contralateral ocular tissues. |
Date |
2023-03 |
References |
None provided. |
Language |
eng |
Format |
application/pdf |
Type |
Text |
Source |
2023 North American Neuro-Ophthalmology Society Annual Meeting |
Relation is Part of |
NANOS Annual Meeting 2023: Scientific Platform Session III: https://novel.utah.edu/collection/NAM/toc/ |
Collection |
Neuro-Ophthalmology Virtual Education Library: NOVEL http://NOVEL.utah.edu |
Publisher |
North American Neuro-Ophthalmology Society |
Holding Institution |
Spencer S. Eccles Health Sciences Library, University of Utah, 10 N 1900 E SLC, UT 84112-5890 |
Rights Management |
Copyright 2023. For further information regarding the rights to this collection, please visit: https://NOVEL.utah.edu/about/copyright |
ARK |
ark:/87278/s67ab6xg |
Setname |
ehsl_novel_nam |
ID |
2389030 |
Reference URL |
https://collections.lib.utah.edu/ark:/87278/s67ab6xg |