Phase I/IIa Visual Acuity Outcomes 1.5-Years Post-Treatment with rAAV2/2-ND4,; Investigational Gene Therapy for ND4 LHON (video)
Creator
Catherine Vignal; Scott Uretsky; Nitza Thomasson; Céline Bouquet; Anne Galy; Jean Philippe Combal; Serge Fitoussi; Jose Aalin Sahel
Affiliation
(CV) Foundation Rothschild, Paris, France; (SU) (NT) (CB) (AG) (JPC) (SF) Gensight Biologics, Paris, France; (JAS) Centre Hospitalier National des Quinze Vingts, Paris, France
Subject
Genetic Disease; Optic Neuropathy
Description
rAAV2/2-ND4 is an experimental gene therapy enabling allotopic transgene expression. We report visual acuity (VA) outcomes 1.5- years post-treatment in a Phase I/IIa (NCT02064569) open-label, dose-escalation safety study.
Date
2017-04-04
Language
eng
Format
video/mp4
Type
Image/MovingImage
Source
2017 North American Neuro-Ophthalmology Society Annual Meeting
