| Identifier |
20160301_nanos_sciplatform2_09 |
| Title |
Recombinant AAV2 Containing the Wild-Type ND4 Gene (rAAV2/2-ND4) is an Experimental Gene Therapy for Vision Loss in LHON Due to the ND4 Mitochondrial Mutation: Phase I/IIa Safety Investigation Results and Upcoming Pivotal Phase III Efficacy Studies (.pdf) |
| Creator |
Catherine Vignal; Scott Uretsky; Nitza Thomasson; Geraldine Honnet; Marisol Corral-Debrinski; Celine Bouquet; Anne Galy; Jean-Philippe Combal; Serge Fitoussi; José A. Sahel |
| Affiliation |
(SV) (JAS) Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts, Paris, France; (CV) (JAS) Fondation Ophtalmologique A de Rothschild, Paris, France; (SU) (NT) (CB) (AG) (JC) (SF) Gensight-Biologics, Paris, France; (GH) Genethon, Evry, France; (MC) Inserm UMR 1141, Paris, France; (JAS) Institut de la Vision, Paris, France |
| Subject |
Optic Neuropathy; Genetic Disease |
| Description |
Allotopic expression of the wild-type ND4 gene delivered by a rAAV2/2 vector through intra-vitreal injection is an experimental therapy for vision loss in patients with ND4 LHON. |
| Date |
2016-03-01 |
| Language |
eng |
| Format |
application/pdf |
| Type |
Text |
| Source |
2016 North American Neuro-Ophthalmology Society Annual Meeting |
| Relation is Part of |
NANOS Annual Meeting 2016: Scientific Platform Session II |
| 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 |
| ARK |
ark:/87278/s67t0vnt |
| Context URL |
The NANOS Annual Meeting Neuro-Ophthalmology Collection: https://novel.utah.edu/collection/NAM/toc/ |
| Setname |
ehsl_novel_nam |
| ID |
185329 |
| Reference URL |
https://collections.lib.utah.edu/ark:/87278/s67t0vnt |
