NOVEL - NANOS Annual Meeting

Safety/Acuity Outcomes 96-Weeks Post-Treatment with rAAV2/2-ND4; Gene Therapy for ND4 LHON: a Phase I/II Trial

Update Item Information
Identifier 20180305_nanos_sciplatform1_08
Title Safety/Acuity Outcomes 96-Weeks Post-Treatment with rAAV2/2-ND4; Gene Therapy for ND4 LHON: a Phase I/II Trial
Creator Barrett Katz; Catherine Vignal; Celine Bouquet; Nitza Thomasson; Ann Galy; Serge Fitoussi; Jose Sahel
Affiliation (BK) Albert Einstein College of Medicine and the Montefiore Medical Center, New York, New York; (CV) (CB) Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts, Paris, France; (BK) (CB) (AG) (SF) GenSight Biologics, Paris, France; (JS)Sorbonne Universités, UPMC Univ Paris 06, Paris, France
Subject Optic Neuropathy; Genetic Disease; LHON; Gene Therapy
Description rAAV2/2-ND4 is an investigational gene therapy enabling allotopic transgene expression. We report safety and visual acuity outcomes 96 weeks post-treatment in a Phase I/II open-label dose escalation trial.
Date 2018-04
Language eng
Format video/mp4
Type Image/MovingImage
Source 2018 North American Neuro-Ophthalmology Society Annual Meeting
Relation is Part of NANOS Annual Meeting 2018: Scientific Platform, Session I
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 2018. For further information regarding the rights to this collection, please visit: https://NOVEL.utah.edu/about/copyright
ARK ark:/87278/s6zh0vn7
Setname ehsl_novel_nam
ID 1316070
Reference URL https://collections.lib.utah.edu/ark:/87278/s6zh0vn7
Back to Search Results