Efficient transduction and optogenetic stimulation of retinal bipolar cells by a synthetic adeno‐associated virus capsid and promoter - Inserm - Institut national de la santé et de la recherche médicale Accéder directement au contenu
Article Dans Une Revue EMBO Molecular Medicine Année : 2014

Efficient transduction and optogenetic stimulation of retinal bipolar cells by a synthetic adeno‐associated virus capsid and promoter

Résumé

In this report, we describe the development of a modified adeno-associated virus (AAV) capsid and promoter for transduction of retinal ON-bipolar cells. The bipolar cells, which are post-synaptic to the photoreceptors, are important retinal targets for both basic and preclinical research. In particular, a therapeutic strategy under investigation for advanced forms of blindness involves using optogenetic molecules to render ON-bipolar cells light-sensitive. Currently, delivery of adequate levels of gene expression is a limiting step for this approach. The synthetic AAV capsid and promoter described here achieves high level of optogenetic transgene expression in ON-bipolar cells. This evokes high-frequency (~100 Hz) spiking responses in ganglion cells of previously blind, rd1, mice. Our vector is a promising vehicle for further development toward potential clinical use.
Fichier principal
Vignette du fichier
emmm0006-1175.pdf (1.56 Mo) Télécharger le fichier
Origine : Publication financée par une institution
Loading...

Dates et versions

inserm-02894121 , version 1 (08-07-2020)

Identifiants

Citer

Thérèse Cronin, Luk H Vandenberghe, Peter Hantz, Josephine Jüttner, Andreas Reimann, et al.. Efficient transduction and optogenetic stimulation of retinal bipolar cells by a synthetic adeno‐associated virus capsid and promoter. EMBO Molecular Medicine, 2014, 6 (9), pp.1175-1190. ⟨10.15252/emmm.201404077⟩. ⟨inserm-02894121⟩

Collections

INSERM
51 Consultations
147 Téléchargements

Altmetric

Partager

Gmail Facebook X LinkedIn More