GPCR Agonist Therapy for Restoring Ciliogenesis in NPHP
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Solution Overview
Problem
There is a need for improved diagnostics and treatments for ciliopathies, particularly nephronophthisis (NPHP), which are characterized by defective primary cilia leading to kidney and retinal anomalies, central nervous system defects, and other abnormalities, with current treatments lacking effectiveness.
Innovation Solution
Administering therapeutically effective amounts of G-protein coupled receptor (GPCR) agonists, such as prostaglandins, to target and restore ciliogenesis in cells affected by ciliopathies, including NPHP, using animal models and patient-derived cell lines to identify effective compounds.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Reliability
If conventional treatments are used for ciliopathies, then current treatment protocols are maintained, but treatment effectiveness is insufficient
Solution Approach 1:
The patent applies parameter changes by modifying cellular signaling parameters through GPCR agonist treatment. Specifically, it changes the activation state of G-protein coupled receptors to restore ciliogenesis in NPHP patient-derived cells, thereby improving treatment effectiveness for ciliopathies where conventional treatments have failed
2Reliability
If GPCR agonists are administered to restore ciliogenesis, then ciliogenesis is restored in affected cells, but the complexity of treatment mechanism increases
Solution Approach 1:
The patent uses G-protein coupled receptors as intermediary molecules to transmit therapeutic effects. The GPCR agonists bind to these receptors, which then mediate the restoration of ciliogenesis through downstream signaling pathways, providing a controlled mechanism to restore cellular function without directly manipulating the complex ciliary assembly processes
Data Source
AI summary
Methods of treating a ciliopathy-associated disease are disclosed, including administering to a subject in need thereof an effective amount of a compound that targets at least one G-protein coupled receptor. Methods for identifying therapeutic agents for treating a disease having a ciliopathy are provided, including providing an animal model system of the ciliopathy for testing a putative therapeutic agent; administering a disruptive agent to the animal, treating the administered animal with the putative therapeutic agent, comparing the measurable phenotype of the treated animal with that of the animal without treatment, and identifying the therapeutic target for treating a ciliopathy, when the measurable phenotype of the treated animal is reduced as compared with that of the animal without treatment.