title: Haematopoietic stem cell gene therapy to treat autoimmune disease.
abstract: Autoimmune diseases affect approximately 6% of the population and are characterised by a pathogenic immune response that targets self-antigens. Well known diseases of this nature include type 1 diabetes, systemic lupus erythematosus, rheumatoid arthritis and multiple sclerosis. Treatment is often restricted to replacement therapy or immunosuppressive regimes and to date there are no cures. The strategy of utilising autologous or allogeneic haematopoietic stem cell transplantation to treat autoimmunity and induce immunological tolerance has been trailed with various levels of success. A major issue is disease relapse as the autoimmune response is reinitiated. Cells of the immune system originate from bone marrow and have a central role in the induction of immunological tolerance. The ability to isolate and genetically manipulate bone marrow haematopoietic stem cells therefore makes these cells a suitable vehicle for driving ectopic expression of defined autoantigens and induction of immunological tolerance.
authors.affiliation: Department of Immunology, Monash University, Commercial Road, Prahran, Victoria 3181, Australia. frank.alderuccio@med.monash.edu.au
authors.forename: Frank
authors.initials: F
authors.lastname: Alderuccio
authors.forename: Christopher
authors.initials: C
authors.lastname: Siatskas
authors.forename: James
authors.initials: J
authors.lastname: Chan
authors.forename: Judith
authors.initials: J
authors.lastname: Field
authors.forename: Kim
authors.initials: K
authors.lastname: Murphy
authors.forename: Zeyad
authors.initials: Z
authors.lastname: Nasa
authors.forename: Ban-Hock
authors.initials: BH
authors.lastname: Toh
mesh_terms: D000818:Animals; D001327:Autoimmune Diseases / Q000235:genetics* / Q000628:therapy; D015551:Autoimmunity; D016026:Bone Marrow Transplantation / Q000276:immunology; D003713:Dendritic Cells / Q000276:immunology; D015316:Genetic Therapy / Q000379:methods*; D018380:Hematopoietic Stem Cell Transplantation; D006412:Hematopoietic Stem Cells / Q000502:physiology*; D006801:Humans; D007108:Immune Tolerance; D051379:Mice; D023421:Models, Animal; D013601:T-Lymphocytes / Q000276:immunology; D014182:Transplantation, Autologous / Q000276:immunology; D014184:Transplantation, Homologous / Q000276:immunology