Alexion Pharmaceuticals, Inc.
METHODS FOR TREATING DISEASES ASSOCIATED WITH CILIOPATHIES

Abstract:

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.