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EMBO Mol Med ; 6(9): 1175-90, 2014 Sep.
Article in English | MEDLINE | ID: mdl-25092770

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.


Subject(s)
Dependovirus/genetics , Retinal Bipolar Cells/virology , Transduction, Genetic/methods , Animals , Genetic Vectors , HEK293 Cells , Humans , Mice , Mice, Inbred C57BL , Mutagenesis, Site-Directed , Promoter Regions, Genetic
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