| Identifier |
20200310_nanos_sciplatform3_05-pdf |
| Title |
Bilateral Visual Improvement with Unilateral Gene Therapy for Leber Hereditary Optic Neuropathy (LHON) (PDF) |
| Creator |
Nancy J. Newman, Mark Moster, Alfredo Sadun, Thomas Klopstock, Catherine Vignal-Clermont, Valerio Carelli, Patrick Yu-Wai-Man, Robert Sergott, Valerie Biousse, Laure Blouin, Magali Taiel, Pierre Burguiere, Caroline Chevalier, Katz Barrett, Jose-Alain Sahel |
| Subject |
Genetic Disease, Optic Neuropathy |
| Description |
LHON is a mitochondrially-inherited disease causing severe bilateral optic neuropathies. A point mutation in the mitochondrial ND4-gene accounts for 75% of LHON cases. rAAV2/2-ND4 is a gene therapy e nabling allotopic expression and delivery of the wildtype ND4 protein to mitochondria of retinal ganglion cells. We assessed the clinical efficacy of a single intravitreal injection of rAAV2/2-ND4 (GS010), an investigational gene therapy for ND4-LHON. |
| Date |
2020-03 |
| Language |
eng |
| Format |
application/pdf |
| Source |
2020 North American Neuro-Ophthalmology Society Annual Meeting |
| Relation is Part of |
NANOS Annual Meeting 2020: Scientific Platform Session III |
| 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 2020. For further information regarding the rights to this collection, please visit: https://NOVEL.utah.edu/about/copyright |
| ARK |
ark:/87278/s6bg7zpn |
| Context URL |
The NANOS Annual Meeting Neuro-Ophthalmology Collection: https://novel.utah.edu/collection/NAM/toc/ |
| Contributor Primary |
Nancy J. Newman, MD |
| Contributor Secondary |
Mark Moster, Alfredo Sadun, Thomas Klopstock, Catherine Vignal-Clermont, Valerio Carelli, Patrick Yu-Wai-Man, Robert Sergott, Valerie Biousse, Laure Blouin, Magali Taiel, Pierre Burguiere, Caroline Chevalier, Katz Barrett, Jose-Alain Sahel |
| Setname |
ehsl_novel_nam |
| ID |
1543307 |
| Reference URL |
https://collections.lib.utah.edu/ark:/87278/s6bg7zpn |
