title: Modeling complement-driven diseases in transgenic mice: Values and limitations.
abstract: Remarkable advances have been made over past decades in understanding the pathogenesis of complement-mediated diseases. This has led to development of new therapies for, and in some cases re-classification of, complement-driven diseases. This success is due to not only insight from human patients but also studies using transgenic animal models. Animal models that mimic human diseases are useful tools to understand the mechanism of disease and develop new therapies but there are also limitations due to species differences in their complement systems. This review provides a summary of transgenic animal models for three human diseases that are at the forefront of anti-complement therapy, paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS) and C3 glomerulopathy (C3G). They are discussed here as examples to highlight the values and limitations of animal modeling in complement-driven diseases.
authors.affiliation: Department of Systems Pharmacology and Translational Therapeutics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, 19104, United States.
authors.forename: Yoshiyasu
authors.initials: Y
authors.lastname: Ueda
authors.affiliation: Department of Systems Pharmacology and Translational Therapeutics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, 19104, United States.
authors.forename: Damodar
authors.initials: D
authors.lastname: Gullipalli
authors.affiliation: Department of Systems Pharmacology and Translational Therapeutics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, 19104, United States. Electronic address: Songwe@mail.med.upenn.edu.
authors.forename: Wen-Chao
authors.initials: WC
authors.lastname: Song
mesh_terms: D000818:Animals; D065766:Atypical Hemolytic Uremic Syndrome / Q000188:drug therapy / Q000209:etiology / Q000378:metabolism; D003167:Complement Activation / Q000276:immunology; D051056:Complement Inactivating Agents / Q000494:pharmacology / Q000627:therapeutic use; D003165:Complement System Proteins / Q000502:physiology*; D004195:Disease Models, Animal; D004198:Disease Susceptibility; D006457:Hemoglobinuria, Paroxysmal / Q000188:drug therapy / Q000209:etiology / Q000378:metabolism; D006801:Humans; D008822:Mice, Transgenic; D009401:Nephrosis / Q000188:drug therapy / Q000209:etiology / Q000378:metabolism
chemical_list: D051056:Complement Inactivating Agents; D003165:Complement System Proteins
keywords: C3G; Complement; PNH; Transgenic mouse; aHUS