Abstract

Type 1 Diabetes Gene therapy is being used with increasing success to treat a growing group of diseases. What about diabetes? Akbarpour et al. used a lentiviral vector to express insulin in liver cells of a mouse model of type 1 diabetes. The therapy induced regulatory T cells specific for insulin and halted immune cell infiltration into the pancreatic islets. Moreover, when gene therapy was combined with a single dose of monoclonal antibody to CD3, it stopped disease progression in diabetic mice. Thus, expressing an autoantigen in liver cells can induce antigen-specific tolerance in autoimmune disease. Sci. Transl. Med. 7 , 289ra81 (2015).

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