title: Gene Therapy for Autoimmune Disease.
abstract: Advances in understanding the immunological and molecular basis of autoimmune diseases have made gene therapy a promising approach to treat the affected patients. Gene therapy for autoimmune diseases aims to regulate the levels of proinflammatory cytokines or molecules and the infiltration of lymphocytes to the effected sites through successful delivery and expression of therapeutic genes in appropriate cells. The ultimate goal of gene therapy is to restore and maintain the immune tolerance to the relevant autoantigens and improve clinical outcomes for patients. Here, we summarize the recent progress in identifying genes responsible for autoimmune diseases and present examples where gene therapy has been applied as treatments or prevention in autoimmune diseases both in animal models and the clinical trials. Discussion on the advantages and pitfalls of gene therapy strategies employed is provided. The intent of this review is to inspire further studies toward the development of new strategies for successful treatment of autoimmune diseases.
authors.affiliation: Division of Rheumatology/Allergy and Clinical Immunology, School of Medicine, University of California, Davis, CA, 95616, USA.
authors.forename: Shang-An
authors.initials: SA
authors.lastname: Shu
authors.affiliation: Division of Rheumatology/Allergy and Clinical Immunology, School of Medicine, University of California, Davis, CA, 95616, USA.
authors.forename: Jinjun
authors.initials: J
authors.lastname: Wang
authors.affiliation: Institute of Biomedical Sciences, Academia Sinica, Taipei, Taiwan, 11529.
authors.forename: Mi-Hua
authors.initials: MH
authors.lastname: Tao
authors.affiliation: Division of Rheumatology/Allergy and Clinical Immunology, School of Medicine, University of California, Davis, CA, 95616, USA. psleung@ucdavis.edu.
authors.forename: Patrick S C
authors.initials: PS
authors.lastname: Leung
mesh_terms: D000818:Animals; D001327:Autoimmune Diseases / Q000235:genetics / Q000276:immunology / Q000628:therapy*; D002465:Cell Movement / Q000235:genetics; D002986:Clinical Trials as Topic; D016207:Cytokines / Q000378:metabolism; D004195:Disease Models, Animal; D015316:Genetic Therapy; D005822:Genetic Vectors / Q000235:genetics; D006801:Humans; D007108:Immune Tolerance; D056747:Immunomodulation / Q000235:genetics; D008214:Lymphocytes / Q000502:physiology*
chemical_list: D016207:Cytokines
keywords: Animal model; Autoimmune disease; Clinical trial; Delivery vectors; Gene therapy