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

Cronin, Therese; Vandenberghe, Luk H; Hantz, Péter; Juttner, Josephine; Reimann, Andreas; Kacsó, Ágota–Enikő; Huckfeldt, Rachel M; Busskamp, Volker; Kohler, Hubertus; Lagali, Pamela S; Roska, Botond; Bennett, Jean

doi:10.15252/emmm.201404077

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

Published Date:

Aug 04 2014

Publisher's site:

http://dx.doi.org/10.15252/emmm.201404077 New Site Icon

Source:

EMBO Mol Med. 2014; 6(9):1175-1190.

[PDF - 1.56 MB]

Details:

Personal Authors:

Cronin, Therese ; Vandenberghe, Luk H ; Hantz, Péter ; Juttner, Josephine ; Reimann, Andreas ; ... More ▼

Keywords:

[+]

Pubmed ID:

25092770

Pubmed Central ID:

PMC4197864

Description:

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.

Document Type:

Journal Article

Collection(s):

CDC Public Access

Funding:

1R24EY019861‐01A1/EY/NEI NIH HHS/United States
8DP1 EY023177/DP/NCCDPHP CDC HHS/United States
DP1 EY023177/EY/NEI NIH HHS/United States
DP1 OD008267/OD/NIH HHS/United States
R24 EY019861/EY/NEI NIH HHS/United States

Supporting Files:

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