Haematopoietic stem cell gene therapy to treat autoimmune disease.
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.
Alderuccio
Frank
F
Department of Immunology, Monash University, Commercial Road, Prahran, Victoria 3181, Australia. frank.alderuccio@med.monash.edu.au
Siatskas
Christopher
C
Chan
James
J
Field
Judith
J
Murphy
Kim
K
Nasa
Zeyad
Z
Toh
Ban-Hock
BH
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