Identifier |
20170404_nanos_sciplatform2_09-abstract |
Title |
Phase I/IIa Visual Acuity Outcomes 1.5-Years Post-Treatment with rAAV2/2-ND4, Investigational Gene Therapy for ND4 LHON (abstract) |
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 |
Language |
eng |
Format |
application/pdf |
Type |
Text |
Source |
2017 North American Neuro-Ophthalmology Society Annual Meeting |
Relation is Part of |
NANOS Annual Meeting 2017: Scientific Platform 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 2013. For further information regarding the rights to this collection, please visit: https://NOVEL.utah.edu/about/copyright |
ARK |
ark:/87278/s69p6vs4 |
Context URL |
The NANOS Annual Meeting Neuro-Ophthalmology Collection: https://novel.utah.edu/collection/NAM/toc/ |
Setname |
ehsl_novel_nam |
ID |
1273017 |
Reference URL |
https://collections.lib.utah.edu/ark:/87278/s69p6vs4 |