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The cGMP Pathway and Inherited Photoreceptor Degeneration: Targets, Compounds, and Biomarkers

1
Institute for Ophthalmic Research, University of Tübingen, Elfriede-Aulhorn-Strasse 5-7, 72076 Tübingen, Germany
2
Biolog Life Science Institute, 28199 Bremen, Germany
*
Author to whom correspondence should be addressed.
These authors contributed equally to this work.
Genes 2019, 10(6), 453; https://doi.org/10.3390/genes10060453
Received: 15 April 2019 / Revised: 5 June 2019 / Accepted: 11 June 2019 / Published: 14 June 2019
(This article belongs to the Special Issue Molecular Therapies for Inherited Retinal Diseases)
Photoreceptor physiology and pathophysiology is intricately linked to guanosine-3’,5’-cyclic monophosphate (cGMP)-signaling. Here, we discuss the importance of cGMP-signaling for the pathogenesis of hereditary retinal degeneration. Excessive accumulation of cGMP in photoreceptors is a common denominator in cell death caused by a variety of different gene mutations. The cGMP-dependent cell death pathway may be targeted for the treatment of inherited photoreceptor degeneration, using specifically designed and formulated inhibitory cGMP analogues. Moreover, cGMP-signaling and its down-stream targets may be exploited for the development of novel biomarkers that could facilitate monitoring of disease progression and reveal the response to treatment in future clinical trials. We then briefly present the importance of appropriate formulations for delivery to the retina, both for drug and biomarker applications. Finally, the review touches on important aspects of future clinical translation, highlighting the need for interdisciplinary cooperation of researchers from a diverse range of fields. View Full-Text
Keywords: retina; cyclic GMP; apoptosis; necrosis; drug delivery systems; translational medicine retina; cyclic GMP; apoptosis; necrosis; drug delivery systems; translational medicine
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Tolone, A.; Belhadj, S.; Rentsch, A.; Schwede, F.; Paquet-Durand, F. The cGMP Pathway and Inherited Photoreceptor Degeneration: Targets, Compounds, and Biomarkers. Genes 2019, 10, 453.

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