Leber Hereditary Optic Neuropathy Gene Therapy Clinical Trial for G11778A Mutation: Serial Pretreatment Evaluation from Baseline to One Year

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Identifier 20110208_nanos_posters_008
Title Leber Hereditary Optic Neuropathy Gene Therapy Clinical Trial for G11778A Mutation: Serial Pretreatment Evaluation from Baseline to One Year
Creator Lam, Byron; Abukhalil, Fawzi; Porciatti, Vittorio; Feuer, William; Morante, Alexis
Subject Leber Hereditary Optic Neuropathy; Gene Therapy
Description The aim of the preparatory phase of the LHON gene therapy clinical trial is to identify and characterize affected patients and carriers for planned gene therapy studies that will utilize "allotopic expression" by delivering copies of normal nuclear-encoded ND4 gene into the nuclei of retinal ganglion cells via an adeno-associated virus vector.
Date 2011-02-08
Language eng
Format application/pdf
Format Creation application/pdf
Type Text
Source 2011 North American Neuro-Ophthalmology Society Annual Meeting
Relation is Part of NANOS 2011: Poster Presentations
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 2011. For further information regarding the rights to this collection, please visit: https://NOVEL.utah.edu/about/copyright
ARK ark:/87278/s6w12cdm
Context URL The NANOS Annual Meeting Neuro-Ophthalmology Collection: https://novel.utah.edu/collection/NAM/toc/
Setname ehsl_novel_nam
ID 181725
Reference URL https://collections.lib.utah.edu/ark:/87278/s6w12cdm
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