Gene Therapy for Leber Congenital Amaurosis

Update Item Information
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: NANOS Annual Meeting Collection: https://novel.utah.edu/collection/nanos-annual-meeting-collection/
Publisher North American Neuro-Ophthalmology Society
Holding Institution Spencer S. Eccles Health Sciences Library, University of Utah
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
Setname ehsl_novel_nam
ID 180926
Reference URL https://collections.lib.utah.edu/ark:/87278/s63r407p
Back to Search Results