Leber Hereditary Optic Neuropathy G11778A Gene Therapy Clinical Trial: Serial Pretreatment Evaluation from Baseline to Two Years

Update item information
Identifier 20120213_nanos_platform_01
Title Leber Hereditary Optic Neuropathy G11778A Gene Therapy Clinical Trial: Serial Pretreatment Evaluation from Baseline to Two Years
Creator Lam, Byron; Guy, John; Abukhalil, Fawzi; Feuer, William; Aroucha, Potyra; Morante, Alexis
Description The preparatory phase of the LHON gene therapy trial aims to characterize affected patients and carriers for the planned gene therapy study that will utilize "allotopic expression," delivering copies of normal nuclear-encoded ND4 gene into the nuclei of retinal ganglion cells via an adeno-associated virus vector. The normal ND4 protein expressed in the cytoplasm is then imported into the mitochondria.
Relation is Part of NANOS 2012: Platform Presentation
Contributor Secondary Gordon, Lynn K; Sadun, Alfredo A
Publisher Spencer S. Eccles Health Sciences Library, University of Utah
Date 2012-02-13
Type Text
Format application/pdf
Format Creation application/pdf
Source 2012 North American Neuro-Ophthalmology Society Annual Meeting
Rights Management Copyright 2012. For further information regarding the rights to this collection, please visit: https://NOVEL.utah.edu/about/copyright
Holding Institution North American Neuro-Ophthalmology Association. NANOS Executive Office 5841 Cedar Lake Road, Suite 204, Minneapolis, MN 55416
Collection Neuro-ophthalmology Virtual Education Library: NOVEL http://NOVEL.utah.edu
Language eng
ARK ark:/87278/s6vt4zmq
Setname ehsl_novel_nam
Date Created 2012-06-28
Date Modified 2021-05-06
ID 181965
Reference URL https://collections.lib.utah.edu/ark:/87278/s6vt4zmq