Gene therapy for type 1 diabetes: a novel approach for targeted treatment of autoimmunity JOURNAL OF CLINICAL INVESTIGATION Creusot, R. J., Fathman, C. G. 2004; 114 (7): 892-894

Abstract

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.

View details for DOI 10.1172/JC1200423168

View details for Web of Science ID 000224223800008

View details for PubMedID 15467826