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Comment
. 2013 May 7;110(19):E1705.
doi: 10.1073/pnas.1303746110. Epub 2013 Apr 3.

Increased levels of gene therapy may not be beneficial in retinal disease

Ellen Townes-Anderson
Comment

Increased levels of gene therapy may not be beneficial in retinal disease

Ellen Townes-Anderson. Proc Natl Acad Sci U S A. .
No abstract available

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Conflict of interest statement

The author declares no conflict of interest.

Figures

Fig. 1.
Fig. 1.
(Left) An intact rod photoreceptor cell. IS, inner segment; N, nucleus; OS, outer segment; T, terminal. (Right) A portion of the inner segment. In retinal disease, opsin, which normally is sequestered in the outer segment, appears on inner segment membranes. If bound by retinal and in the presence of light, opsin activates an abnormal signaling pathway involving adenylyl cyclase (AC) and leading to increased cAMP, neuritic sprouting, and cell death. Modified from (3), Copyright Association for Research in Vision and Ophthalmology.
See this image and copyright information in PMC

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References

    1. Cideciyan AV, et al. Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement. Proc Natl Acad Sci USA. 2013;110(6):E517–E525. - PMC - PubMed
    1. Alfinito PD, Townes-Anderson E. Activation of mislocalized opsin kills rod cells: A novel mechanism for rod cell death in retinal disease. Proc Natl Acad Sci USA. 2002;99(8):5655–5660. - PMC - PubMed
    1. Wang J, Zhang N, Beuve A, Townes-Anderson E. Mislocalized opsin and cAMP signaling: A mechanism for sprouting by rod cells in retinal degeneration. Invest Ophthalmol Vis Sci. 2012;53(10):6355–6369. - PMC - PubMed
    1. Bonilha VL, et al. Histopathology and functional correlations in a patient with a mutation in RPE65, the gene for retinol isomerase. Invest Ophthalmol Vis Sci. 2011;52(11):8381–8392. - PMC - PubMed
    1. Hernández M, Pearce-Kelling SE, Rodriguez FD, Aguirre GD, Vecino E. Altered expression of retinal molecular markers in the canine RPE65 model of Leber congenital amaurosis. Invest Ophthalmol Vis Sci. 2010;51(12):6793–6802. - PMC - PubMed

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