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
video/mp4
Type
Image/MovingImage
Source
49th Annual North American Neuro-Ophthalmology Society Annual Meeting
