Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Review
. 2012 May 9;32(19):6415-20.
doi: 10.1523/JNEUROSCI.0295-12.2012.

Emerging gene therapies for retinal degenerations

Constance L Cepko  1
Affiliations
Review

Emerging gene therapies for retinal degenerations

Constance L Cepko. J Neurosci. .
No abstract available

PubMed Disclaimer

Figures

Figure 1.
Figure 1.
Targeted cell types and types of genes to be delivered using gene therapy for RP. A, The cell types of the normal retina, with the associated support cells, the retinal pigmented epithelium (RPE) cells. Note that there are many more rods than cones in the photoreceptor layer, approximately 20:1, in both the mouse and the extramacular region of the human retina. In addition, note that the retinal pigmented epithelium processes surround the rod and cone outer segments, providing various types of support functions. The outer segments are membrane-rich and are the location of the phototransduction process. B, The rods typically die first in RP, with a concomitant collapse of the outer segment layer, and a loss of the normal association between the retinal pigmented epithelium processes and the remaining outer segments. Cones exhibit a dramatic change in morphology, modeled here after observations in mice (Lin et al., 2009). The cell types that are being targeted for gene therapy using AAV vectors are shown. For a description of the types of genes being delivered, see Table 1 and the text. HC, horizontal cell; BP, bipolar cells; AC, amacrine cells; MG, Mueller glia; RGC, retinal ganglion cells; ON BP, bipolar cells that hyperpolarize in response to glutamate when light is on. Drawing by Santiago Rompani and C. Cepko.
See this image and copyright information in PMC

References

    1. Bainbridge JW, Smith AJ, Barker SS, Robbie S, Henderson R, Balaggan K, Viswanathan A, Holder GE, Stockman A, Tyler N, Petersen-Jones S, Bhattacharya SS, Thrasher AJ, Fitzke FW, Carter BJ, Rubin GS, Moore AT, Ali RR. Effect of gene therapy on visual function in Leber's congenital amaurosis. N Engl J Med. 2008;358:2231–2239. - PubMed
    1. Bamann C, Nagel G, Bamberg E. Microbial rhodopsins in the spotlight. Curr Opin Neurobiol. 2010;20:610–616. - PubMed
    1. Bermingham-McDonogh O, Reh TA. Regulated reprogramming in the regeneration of sensory receptor cells. Neuron. 2011;71:389–405. - PMC - PubMed
    1. Berson E. Retinal degenerations: planning for the future. Adv Exp Med Biol. 2008;613:21–35. - PubMed
    1. Bi A, Cui J, Ma YP, Olshevskaya E, Pu M, Dizhoor AM, Pan ZH. Ectopic expression of a microbial-type rhodopsin restores visual responses in mice with photoreceptor degeneration. Neuron. 2006;50:23–33. - PMC - PubMed

Publication types

MeSH terms

Substances