rAAV2/2-ND4 Treatment of Leber Optic Neuropathy: 72-Week Data from the REVERSE Phase III Clinical Trial

Update item information
Identifier 20190319_nanos_sciplatform3_01_slides
Title rAAV2/2-ND4 Treatment of Leber Optic Neuropathy: 72-Week Data from the REVERSE Phase III Clinical Trial
Creator Nancy Newman, Mark Moster, Alfredo Sadun, Thomas Klopstock, Catherine Vignal, Valerio Carelli, Patrick Yu-Wai-Man, Jose Sahel, Barrett Katz
Subject Genetic Disease, Optic europathy
Description LHON is a mitochondrially inherited disease that causes bilateral central vision loss. A point mutation in the mitochondrial ND4gene at nucleotide position 11778 accounts for 75% of LHON cases. rAAV2/2-ND4is a gene therapy enabling allotopic expression and delivery of the wildtype ND4 protein to mitochondria within retinal ganglion cells. The clinical efficacy of rAAV2/2-ND4 (GS010) is currently being assessed in several Phase 3 trials of ND4‑LHON subjects.
Relation is Part of NANOS 2019: Scientific Platform III
Contributor Primary Nancy Newman
Contributor Secondary Mark Moster, Alfredo Sadun, Thomas Klopstock, Catherine Vignal, Valerio Carelli, Patrick Yu-Wai-Man, Jose Sahel, Barrett Katz
Publisher Spencer S. Eccles Health Sciences Library, University of Utah
Date 2019-03
Format application/pdf
Source 2019 North American Neuro-Ophthalmology Society Annual Meeting
Rights Management Copyright 2019. 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/s6wh79tw
Setname ehsl_novel_nam
Date Created 2019-09-10
Date Modified 2019-09-10
ID 1455033
Reference URL https://collections.lib.utah.edu/ark:/87278/s6wh79tw
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