title: An autoimmune disease prevented by anti-retroviral drugs.
abstract: BACKGROUND
  Both Aicardi-Goutières syndrome, a Mendelian mimic of congenital infection, and the autoimmune disease systemic lupus erythematosus can result from mutations in the gene encoding the enzyme Trex1. In mice, the absence of Trex1 causes severe myocarditis. The enzyme is thought to degrade endogenous retroelements, thus linking them to autoimmune disease. However, inhibition of reverse transcription by the inhibitor zidovudine (AZT) did not ameliorate the disease, weakening the link to retroelements.
  
  
  RESULTS
  Here, we show that two other FDA-approved drugs that inhibit reverse transcriptase can ameliorate the myocarditis in Trex1-null mouse.
  
  
  CONCLUSIONS
  The result suggests that retroelements contribute to this hereditary form of autoimmunity, and that treatment with retroelement inhibitors might ameliorate Aicardi-Goutières syndrome in humans.
authors: Department of Microbiology and Immunology, University of California, San Francisco, CA 94143-0414, USA. mutator@ucsf.edu
authors: Gabriele B
authors: GB
authors: Beck-Engeser
authors: Dan
authors: D
authors: Eilat
authors: Matthias
authors: M
authors: Wabl
mesh_terms: D000818:Animals; D001327:Autoimmune Diseases / Q000188:drug therapy / Q000235:genetics / Q000276:immunology; D020274:Autoimmune Diseases of the Nervous System / Q000235:genetics / Q000276:immunology; D003841:Deoxycytidine / Q000031:analogs & derivatives* / Q000494:pharmacology; D004338:Drug Combinations; D000069480:Emtricitabine, Tenofovir Disoproxil Fumarate Drug Combination; D005090:Exodeoxyribonucleases / Q000235:genetics*; D049452:Green Fluorescent Proteins / Q000096:biosynthesis; D008180:Lupus Erythematosus, Systemic / Q000235:genetics / Q000276:immunology; D051379:Mice; D018345:Mice, Knockout; D009205:Myocarditis / Q000188:drug therapy* / Q000276:immunology / Q000517:prevention & control*; D009421:Nervous System Malformations / Q000235:genetics / Q000276:immunology; D019829:Nevirapine / Q000494:pharmacology*; D009943:Organophosphorus Compounds / Q000494:pharmacology*; D010750:Phosphoproteins / Q000235:genetics*; D018626:Retroelements; D018894:Reverse Transcriptase Inhibitors / Q000494:pharmacology; D015215:Zidovudine / Q000494:pharmacology
chemical_list: D004338:Drug Combinations; D000069480:Emtricitabine, Tenofovir Disoproxil Fumarate Drug Combination; D009943:Organophosphorus Compounds; D010750:Phosphoproteins; D018626:Retroelements; D018894:Reverse Transcriptase Inhibitors; D003841:Deoxycytidine; D049452:Green Fluorescent Proteins; D015215:Zidovudine; D019829:Nevirapine; D005090:Exodeoxyribonucleases; C429526:three prime repair exonuclease 1