Gene Therapy for Leber Congenital Amaurosis

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Identifier 20070212_nanos_novelnotreatmentsympos_07.pdf
Title Gene Therapy for Leber Congenital Amaurosis
Creator William Hauswirth; Jijing Pang; Sanford Boye
Affiliation University of Florida, Gainesville, FL
Subject Gene Therapy; Leber Congenital Amaurosis; RPE65; AAV Vector; Clinical Trial
Description The first AAV-mediated RPE gene therapy to restore visual function was obtained in a canine model of Leber Congenital Amaurosis (LCA), a severe form of autosomal recessive, childhood-onset retinal dystrophy 1;2. Humans with LCA are born with significant vision deficits and usually lose all vision within ten years.
Date 2007-02-12
Language eng
Format application/pdf
Type Text
Source 2007 North American Neuro-Ophthalmology Society Annual Meeting
Relation is Part of NANOS 2007: Novel Approaches to Treatment of Neuro-Ophthalmic Diseases: Part II: Neuroprotection and Other Methods for Salvaging Visual Function
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 2010. For further information regarding the rights to this collection, please visit: https://NOVEL.utah.edu/about/copyright
ARK ark:/87278/s63r407p
Context URL The NANOS Annual Meeting Neuro-Ophthalmology Collection: https://novel.utah.edu/collection/NAM/toc/
Setname ehsl_novel_nam
ID 180926
Reference URL https://collections.lib.utah.edu/ark:/87278/s63r407p
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