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Long-term expression of melanopsin and channelrhodopsin causes no gross alterations in the dystrophic dog retina

Authors :
Kizito-Tshitoko Tshilenge
Lyse Libeau
Philippe Moullier
Cronin T
Michel Weber
Pichard
Baptiste Ameline
Serge Picaud
Carolina Isiegas
Nathalie Provost
Caroline Guihal
Alexandra Mendes-Madeira
Marine Biget
Romain Caplette
Cronin, Therese
Laboratoire de Thérapie Génique Translationnelle des Maladies Génétiques (Inserm UMR 1089)
Institut National de la Santé et de la Recherche Médicale (INSERM)-Université de Nantes - UFR de Médecine et des Techniques Médicales (UFR MEDECINE)
Université de Nantes (UN)-Université de Nantes (UN)
Service d'ophtalmologie [Nantes]
Hôtel-Dieu
Institut de la Vision
Université Pierre et Marie Curie - Paris 6 (UPMC)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS)
Génomique Environnementale et Humaine (GEH)
Université de Rennes 1 (UR1)
Université de Rennes (UNIV-RENNES)-Université de Rennes (UNIV-RENNES)
Vecteurs viraux et transfert des gènes in vivo
Institut National de la Santé et de la Recherche Médicale (INSERM)
Source :
Gene Therapy, Gene Therapy, 2017, 24 (11), pp.735-741. ⟨10.1038/gt.2017.63⟩, Gene Therapy, Nature Publishing Group, 2017, 24 (11), pp.735-741. ⟨10.1038/gt.2017.63⟩
Publication Year :
2017
Publisher :
HAL CCSD, 2017.

Abstract

International audience; Several preclinical studies have investigated the potential of algal channelrhodopsin and human melanopsin as optogenetic tools for vision restoration. In the present study, we assessed the potentially deleterious effects of long-term expression of these optogenes on the diseased retina in a large animal model of retinal degeneration, the RPE65-deficient Briard dog model of Leber congenital amaurosis. Intravitreal injection of adeno-associated virus vectors expressing channelrhodopsin and melanopsin had no effect on retinal thickness over a 16-month period post injection. Our data support the safety of the optogenetic approach for the treatment of blindness.

Details

Language :
English
ISSN :
09697128 and 14765462
Database :
OpenAIRE
Journal :
Gene Therapy, Gene Therapy, 2017, 24 (11), pp.735-741. ⟨10.1038/gt.2017.63⟩, Gene Therapy, Nature Publishing Group, 2017, 24 (11), pp.735-741. ⟨10.1038/gt.2017.63⟩
Accession number :
edsair.doi.dedup.....795340e3090ab3ee878622bfb73af08a
Full Text :
https://doi.org/10.1038/gt.2017.63⟩