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

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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
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.
Relation is Part of NANOS Annual Meeting 2018: Scientific Platform, Session I
Contributor Primary Barrett Katz
Contributor Secondary Catherine Vignal, Celine Bouquet, Nitza Thomasson, Ann Galy, Serge Fitoussi, Jose Sahel
Publisher Spencer S. Eccles Health Sciences Library, University of Utah
Date 2018-04
Format video/mp4
Source 2018 North American Neuro-Ophthalmology Society Annual Meeting
Rights Management Copyright 2018. For further information regarding the rights to this collection, please visit: https://NOVEL.utah.edu/about/copyright
Collection Neuro-ophthalmology Virtual Education Library: NOVEL http://NOVEL.utah.edu
Language eng
ARK ark:/87278/s6zh0vn7
Setname ehsl_novel_nam
Date Created 2018-04-25
Date Modified 2018-04-25
ID 1316070
Reference URL https://collections.lib.utah.edu/ark:/87278/s6zh0vn7
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