title: Humanized mouse models for organ-specific autoimmune diseases.
abstract: Murine models for human autoimmune diseases are an essential tool for studying pathogenesis and for identifying new therapeutic targets. Mice are not the natural disease host, and conventional models have proved to be poor predictors of efficacy and safety in recent trials aiming to translate drug and biologic treatments to humans. Evidently, further steps towards recapitulating human diseases are urgently needed, for example using transgenic predisposing human HLA allele(s) plus T-cell receptor(s) implicated in a representative patient's autoimmune disease. The latest development - humanizing most of the immune system by transplanting human hematopoietic stem cells into severely immunodeficient mice - should lead to even better modeling.
authors.affiliation: MRC Human Immunology Unit and Department of Clinical Neurology, Weatherall Institute of Molecular Medicine, John Radcliffe Hospital, University of Oxford, Oxford OX3 9DS, United Kingdom.
authors.forename: Manuel A
authors.initials: MA
authors.lastname: Friese
authors.forename: Lise T
authors.initials: LT
authors.lastname: Jensen
authors.forename: Nick
authors.initials: N
authors.lastname: Willcox
authors.forename: Lars
authors.initials: L
authors.lastname: Fugger
mesh_terms: D000818:Animals; D001327:Autoimmune Diseases; D004195:Disease Models, Animal; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D051379:Mice; D008822:Mice, Transgenic / Q000276:immunology*