Preventing autoimmune diabetes through gene therapy.
Extract: Type 1 diabetes is an autoimmune disease in which an individual develops T cells that are able to destroy insulin-producing beta cells in the pancreas. Type 1 diabetics require life-long treatment with exogenous insulin for survival. Susceptibility to type 1 diabetes is influenced by both genetic and environmental factors. The first diabetes-susceptibility genes to be identified were the human leukocyte antigen (HLA) genes. Subsequent studies demonstrated an association of these genes with the insulin gene region. High throughput screening of the human genome in families with two or more affected siblings led to the identification of additional chromosomal regions that may contain susceptibility genes for type 1 diabetes. However, linkage between the HLA gene region and susceptibility to disease suggested that the principal genetic component leading to development of diabetes is the inheritance of mutant HLA class II alleles. These are so-called "at-risk" alleles which lack an aspartic acid, a positively amino acid, at position 57 of the major histocompatibility complex (MHC) class II beta chain.
Tian
Chaorui
C
Transplantation Research Center, Brigham and Women's Hospital and Children's Hospital Boston, Harvard Medical School, Boston, MA 02115, USA.
Bagley
Jessamyn
J
Iacomini
John
J