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

Back to Previous Page

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

Source:

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

[PDF-1.56 MB]

Details:

Alternative Title:

EMBO Mol Med

Personal Author:

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

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.

Subject:

[+]

Pubmed ID:

25092770

Pubmed Central ID:

PMC4197864

Document Type:

Journal Article

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 ;

Collection(s):

CDC Public Access

Main Document Checksum:

[+]

File Type:

Supporting Files:

	emmm0006-1175-SD1.pdf	application/pdf
	emmm0006-1175-SD2.pdf	application/pdf
	emmm0006-1175-SD3.pdf	application/pdf
	emmm0006-1175.nxml	text/plain
	license.txt	text/plain
	emmm0006-1175-f1.gif	image/gif
	emmm0006-1175-f1.jpg	image/jpeg
	emmm0006-1175-f2.gif	image/gif
	emmm0006-1175-f2.jpg	image/jpeg
	emmm0006-1175-f3.gif	image/gif
	emmm0006-1175-f3.jpg	image/jpeg
	emmm0006-1175-f4.gif	image/gif
	emmm0006-1175-f4.jpg	image/jpeg

No Related Documents.

Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain

[PDF - 2.06 MB]

Personal Author:

Deverman, Benjamin E. ; Pravdo, Piers L. ; ...

Published Date:

Feb 1 2016

Source:

Nat Biotechnol. 34(2):204-209.

Description:

Recombinant adeno-associated viruses (rAAVs) are commonly used vehicles for in vivo gene transfer. However, the tropism repertoire of naturally occurr...

File Type:

[PDF - 2.06 MB]

File Type:

[PDF - 2.06 MB]
Locus-specific editing of histone modifications at endogenous enhancers using programmable TALE-LSD1 fusions

[PDF - 1.14 MB]

Personal Author:

Mendenhall, Eric M. ; Williamson, Kaylyn E. ; ...

Published Date:

Sep 08 2013

Source:

Nat Biotechnol. 31(12):1133-1136.

Description:

Mammalian gene regulation is dependent on tissue-specific enhancers that can act across large distances to influence transcriptional activity. Mapping...

File Type:

[PDF - 1.14 MB]

File Type:

[PDF - 1.14 MB]
Differential targeting of feline photoreceptors by recombinant adeno-associated viral vectors: implications for preclinical gene therapy trials

[PDF - 2.09 MB]

Personal Author:

Minella, AL ; Mowat, FM ; ...

Published Date:

Jul 24 2014

Source:

Gene Ther. 2014; 21(10):913-920.

Description:

The cat is emerging as a promising large animal model for preclinical testing of retinal dystrophy therapies, for example, by gene therapy. However, t...

File Type:

[PDF - 2.09 MB]

File Type:

[PDF - 2.09 MB]
Identification of novel small molecule modulators of K2P18.1 two-pore potassium channel

[PDF - 862.46 KB]

Personal Author:

Bruner, J. Kyle ; Zou, Beiyan ; ...

Published Date:

Jun 24 2014

Source:

Eur J Pharmacol. 2014; 740:603-610

Description:

Two-pore domain potassium (K2P) channels are responsible for background potassium (K+) current, which is crucial for the maintenance of resting membra...

File Type:

[PDF - 862.46 KB]

File Type:

[PDF - 862.46 KB]
Optical Control of Mammalian Endogenous Transcription and Epigenetic States

[PDF - 1.06 MB]

Personal Author:

Konermann, Silvana ; Brigham, Mark D. ; ...

Published Date:

Aug 23 2013

Source:

Nature. 500(7463):472-476.

Description:

The dynamic nature of gene expression enables cellular programming, homeostasis and environmental adaptation in living systems. Dissection of causal g...

File Type:

[PDF - 1.06 MB]

File Type:

[PDF - 1.06 MB]

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

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

You May Also Like:

Have Questions?

CDC INFORMATION

CONNECT WITH CDC