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

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

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

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Citation: Cronin, T., L. H. Vandenberghe, P. Hantz, J. Juttner, A. Reimann, Á. Kacsó, R. M. Huckfeldt, et al. 2014. “Efficient transduction and optogenetic stimulation of retinal bipolar cells by a synthetic adeno-associated virus capsid and promoter.” EMBO Molecular Medicine 6 (9): 1175-1190. doi:10.15252/emmm.201404077. http://dx.doi.org/10.15252/emmm.201404077.
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Abstract: 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.
Published Version: doi:10.15252/emmm.201404077
Other Sources: http://www.ncbi.nlm.nih.gov/pmc/articles/PMC4197864/pdf/
Terms of Use: This article is made available under the terms and conditions applicable to Other Posted Material, as set forth at http://nrs.harvard.edu/urn-3:HUL.InstRepos:dash.current.terms-of-use#LAA
Citable link to this page: http://nrs.harvard.edu/urn-3:HUL.InstRepos:13454822
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