title: Hematopoietic stem cell gene therapy as a treatment for autoimmune diseases.
abstract: A key function of the immune system is to protect us from foreign pathogens such as viruses, bacteria, fungi and multicellular parasites. However, it is also important in many other aspects of human health such as cancer surveillance, tissue transplantation, allergy and autoimmune disease. Autoimmunity can be defined as a chronic immune response that targets self-antigens leading to tissue pathology and clinical disease. Autoimmune diseases, as a group of diseases that include type 1 diabetes, multiple sclerosis, rheumatoid arthritis and systemic lupus erythematosus, have no effective cures, and treatment is often based on long-term broad-spectrum immunosuppressive regimes. While a number of strategies aimed at providing disease specific treatments are being explored, one avenue of study involves the use of hematopoietic stem cells to promote tolerance. In this manuscript, we will review the literature in this area but in particular examine the relatively new experimental field of gene therapy and hematopoietic stem cell transplantation as a molecular therapeutic strategy to combat autoimmune disease.
authors: Department of Immunology, Monash Central Clinical School, Monash University, Victoria, Australia. frank.alderuccio@monash.edu
authors: Frank
authors: F
authors: Alderuccio
authors: Zeyad
authors: Z
authors: Nasa
authors: Jieyu
authors: J
authors: Chung
authors: Hyun-Ja
authors: HJ
authors: Ko
authors: James
authors: J
authors: Chan
authors: Ban-Hock
authors: BH
authors: Toh
mesh_terms: D000818:Animals; D001327:Autoimmune Diseases / Q000276:immunology / Q000517:prevention & control / Q000628:therapy*; D002478:Cells, Cultured / Q000637:transplantation; D003131:Combined Modality Therapy; D004681:Encephalomyelitis, Autoimmune, Experimental / Q000276:immunology / Q000517:prevention & control / Q000628:therapy; D018014:Gene Transfer Techniques; D015316:Genetic Therapy; D018380:Hematopoietic Stem Cell Transplantation; D006412:Hematopoietic Stem Cells / Q000276:immunology; D006801:Humans; D007165:Immunosuppression Therapy / Q000379:methods*; D051379:Mice