Gene therapy for type 1 diabetes: a novel approach for targeted treatment of autoimmunity.
It has been difficult to develop therapies that target those T cells initiating and mediating the pathogenesis of autoimmune disease. Indeed, most current treatments indiscriminately affect both the autoreactive T cells and the "good" T cells, putting the patient at risk of compromised immune function. A new approach raises the possibility of targeted therapy for autoimmunity. Transplantation of hematopoietic stem cells modified to express a protective form of MHC class II corrects a defect in central tolerance. This method contrasts with other targeted therapies that attempt to modify peripheral tolerance, which is also defective in type 1 diabetes mellitus.
Department of Medicine, Division of Immunology and Rhematology, Stanford University School of Medicine, Stanford, Califormia 94305, USA.
Rémi J
RJ
Creusot
C Garrison
CG
Fathman
D000818:Animals; D015551:Autoimmunity / Q000235:genetics*; D003922:Diabetes Mellitus, Type 1 / Q000235:genetics* / Q000276:immunology / Q000628:therapy*; D015316:Genetic Therapy; D006801:Humans; D051379:Mice; D016688:Mice, Inbred NOD; D016176:T-Lymphocyte Subsets; D013601:T-Lymphocytes / Q000276:immunology / Q000502:physiology