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Journal articles
Efficient transduction and optogenetic stimulation of retinal bipolar cells by a synthetic adeno‐associated virus capsid and promoter
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.
Cited literature [59 references]
https://www.hal.inserm.fr/inserm-02894121
Contributor : Therese Cronin <>
Submitted on : Wednesday, July 8, 2020 - 5:35:27 PM
Last modification on : Thursday, July 9, 2020 - 11:37:39 AM
Contributor : Therese Cronin <>
Submitted on : Wednesday, July 8, 2020 - 5:35:27 PM
Last modification on : Thursday, July 9, 2020 - 11:37:39 AM
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emmm0006-1175.pdf
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