| 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 |
| Affiliation |
(NJN) (VB) Emory University School of Medicine, Atlanta, GA; (MM) (RS) Wills Eye Hospital and Thomas Jefferson University, Philadelphia, PA; (AS) Doheny Eye Institute/UCLA School of Medicine, Los Angeles, CA; (TK) Friedrich-Baur-Institute, Ludwig-Maximilians-University, Munich, Germany; (CVC) Centre Hospitalier National d'Ophtalmologie des Quinze Vingts, Paris, France; (VC) IRCCS Istituto delle Scienze Neurologiche di Bologna, UOC Clinica Neurologica, Bologna, Italy; (PYWM) Cambridge Centre for Brain Repair, Cambridge U; Moorfields Eye Hospital, Cambridge and London, United Kingdom of Great Britain and Northern Ireland; (LB) (MT) (PB) (CC) (KB) GenSight Biologics, Paris, France; (JAS) Institut de la Vision; University of Pittsburgh School of Medicine, Pittsburgh and Paris, Pennsylvania, France |
| 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 enabling 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 |
| Type |
Text |
| 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/ |
| Setname |
ehsl_novel_nam |
| ID |
1543307 |
| Reference URL |
https://collections.lib.utah.edu/ark:/87278/s6bg7zpn |
