| Identifier |
20180305_nanos_sciplatform1_08-1 |
| Title |
Safety/Acuity Outcomes 96-Weeks Post-Treatment with rAAV2/2-ND4; Gene Therapy for ND4 LHON: a Phase I/II Trial |
| Creator |
Barrett Katz; Catherine Vignal; Celine Bouquet; Nitza Thomasson; Ann Galy; Serge Fitoussi; Jose Sahel |
| Affiliation |
(BK) Albert Einstein College of Medicine and the Montefiore Medical Center, New York, New York; (CV) (CB) Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts, Paris, France; (BK) (CB) (AG) (SF) GenSight Biologics, Paris, France; (JS)Sorbonne Universités, UPMC Univ Paris 06, Paris, France |
| Subject |
Optic Neuropathy; Genetic Disease; LHON; Gene Therapy |
| Description |
rAAV2/2-ND4 is an investigational gene therapy enabling allotopic transgene expression. We report safety and visual acuity outcomes 96 weeks post-treatment in a Phase I/II open-label dose escalation trial. |
| Date |
2018-04 |
| Language |
eng |
| Format |
application/pdf |
| Type |
Text |
| Source |
2018 North American Neuro-Ophthalmology Society Annual Meeting |
| Relation is Part of |
NANOS Annual Meeting 2018: Scientific Platform, Session I |
| 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 2018. For further information regarding the rights to this collection, please visit: https://NOVEL.utah.edu/about/copyright |
| ARK |
ark:/87278/s6tr0406 |
| Setname |
ehsl_novel_nam |
| ID |
1316071 |
| Reference URL |
https://collections.lib.utah.edu/ark:/87278/s6tr0406 |
