| Identifier |
20190319_nanos_sciplatform3_01_slides |
| Title |
rAAV2/2-ND4 Treatment of Leber Optic Neuropathy: 72-Week Data from the REVERSE Phase III Clinical Trial |
| Creator |
Nancy J. Newman; Mark Moster; Alfredo Sadun; Thomas Klopstock; Catherine Vignal; Valerio Carelli; Patrick Yu-Wai-Man; Jose Sahel; Barrett Katz |
| Affiliation |
(NJN) Emory University School of Medicine, Atlanta, GA; (MM) Wills Eye Hospital, Sidney Kimmel Medical College of Thomas Jefferson University, Philadelphia, PA; (AS) Doheny Eye Institute and Department of Ophthalmology, UCLA, Los Angeles, CA; (TK) Friedrich-Baur-Institute, University Hospital of LMU, Munich, Germany; (CV) CHNO Quinze-Vingts and Fondation Ophtalmologique Rothschild, Paris, France; (VC) University of Bologna, Bologna, Italy; (PYWM) Moorfields Eye Hospital, UCL Institute of Ophthalmology, University of Cambridge, London and Cambridge, United Kingdom of Great Britain and Northern Ireland; (JS) University of Pittsburgh and CNO 15/20 in Paris, France, Pittsburgh, PA; (BK) GenSight, Paris, France |
| Subject |
Genetic Disease; Optic Europathy |
| Description |
LHON is a mitochondrially inherited disease that causes bilateral central vision loss. A point mutation in the mitochondrial ND4gene at nucleotide position 11778 accounts for 75% of LHON cases. rAAV2/2-ND4is a gene therapy enabling allotopic expression and delivery of the wildtype ND4 protein to mitochondria within retinal ganglion cells. The clinical efficacy of rAAV2/2-ND4 (GS010) is currently being assessed in several Phase 3 trials of ND4‑LHON subjects. |
| Date |
2019-03 |
| Language |
eng |
| Format |
application/pdf |
| Format Creation |
Microsoft PowerPoint |
| Type |
Text |
| Source |
2019 North American Neuro-Ophthalmology Society Annual Meeting |
| Relation is Part of |
NANOS 2019: Scientific Platform III |
| 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 2019. For further information regarding the rights to this collection, please visit: https://NOVEL.utah.edu/about/copyright |
| ARK |
ark:/87278/s6wh79tw |
| Setname |
ehsl_novel_nam |
| ID |
1455033 |
| Reference URL |
https://collections.lib.utah.edu/ark:/87278/s6wh79tw |
