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Cyclic nucleotide signaling as a drug target in retinitis pigmentosa

Katri Vainionpää1, Ahmed B Montaser1, Henri Leinonen1

  • 1School of Pharmacy, Faculty of Health Sciences, University of Eastern Finland, Kuopio, Finland.

FEBS Letters
|July 9, 2025
PubMed

Insights

Targeting cyclic nucleotide signaling pathways offers a promising mutation-independent treatment for retinitis pigmentosa (RP). This approach could slow disease progression, benefiting a wide range of RP patients.

Area of Science:

  • Ophthalmology
  • Genetics
  • Pharmacology

Background:

  • Retinitis pigmentosa (RP) is a diverse inherited retinal disease with over 90 causative genes.
  • Genetic complexity and cost hinder gene therapy applications for RP.
  • A universal pharmacological strategy is needed to slow RP progression irrespective of genetic cause.

Purpose of the Study:

  • To propose cyclic nucleotide signaling systems as druggable targets for retinitis pigmentosa.
  • To explore mutation-independent therapeutic strategies for RP.

Main Methods:

  • Review of the role of cyclic guanosine monophosphate (cGMP) and cyclic adenosine monophosphate (cAMP) in retinal function.
  • Analysis of evidence supporting the inhibition of cGMP and cAMP signaling in retinal degeneration models.

Main Results:

  • Cyclic nucleotide signaling is crucial for retinal phototransduction.
  • Dysregulation of cGMP and cAMP pathways is linked to retinal degeneration.
  • Inhibiting these pathways has shown therapeutic potential in preclinical models.

Conclusions:

  • Targeting cyclic nucleotide signaling (cGMP and cAMP) presents a viable mutation-independent therapeutic strategy for RP.
  • This approach may enable broad disease modification through drug repurposing and advanced delivery systems.
Keywords:
cAMPcGMPcyclic nucleotidesretinal degenerationretinitis pigmentosa

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