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

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Identifier 20190319_nanos_sciplatform3_01-video
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.
Date 2019-03
Language eng
Format video/mp4
Source 2019 North American Neuro-Ophthalmology Society Annual Meeting
Relation is Part of NANOS 2019: Scientific Platform III
Collection North American Neuro-Ophthalmology Association. NANOS Executive Office 5841 Cedar Lake Road, Suite 204, Minneapolis, MN 55416
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 2019. For further information regarding the rights to this collection, please visit: https://NOVEL.utah.edu/about/copyright
ARK ark:/87278/s6p31fgs
Context URL The NANOS Annual Meeting Neuro-Ophthalmology Collection: https://novel.utah.edu/collection/NAM/toc/
Contributor Primary Nancy Newman
Contributor Secondary Mark Moster, Alfredo Sadun, Thomas Klopstock, Catherine Vignal, Valerio Carelli, Patrick Yu-Wai-Man, Jose Sahel, Barrett Katz
Setname ehsl_novel_nam
ID 1427558
Reference URL https://collections.lib.utah.edu/ark:/87278/s6p31fgs
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