{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"submitter":["Crow YJ"],"funding":["Medical Research Council","National Institute for Health Research (NIHR)"],"pagination":["750-757"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC7617231"],"repository":["biostudies-literature"],"omics_type":["Unknown"],"volume":["67(6)"],"pubmed_abstract":["<h4>Aim</h4>To extend the findings of a previous clinical trial suggesting combined abacavir (ABC), lamivudine (3TC), and zidovudine (AZT) reduces type I interferon (IFN) signalling in Aicardi-Goutières syndrome (AGS).<h4>Method</h4>This was an open label, non-placebo-controlled phase II clinical trial (NCT04731103) in patients less than 16 years with any of five AGS genotypes. The effect of ABC or 3TC individually, or of combined ABC + 3TC + AZT, on IFN-stimulated gene (ISG) expression (primary outcome) and IFN-alpha protein (secondary outcome) in blood was assessed.<h4>Results</h4>Thirteen patients were recruited. Compliance was poor in the ABC + 3TC + AZT arm. No statistically significant effects were observed with ABC or 3TC, or with ABC + 3TC + AZT over 6 weeks. A statistically signif"],"journal":["Developmental medicine and child neurology"],"pubmed_title":["Reverse transcriptase inhibitors in Aicardi-Goutieres syndrome: A crossover clinical trial."],"pmcid":["PMC7617231"],"funding_grant_id":["MC_UU_00035/11","NIHR203956","MR/S034676/1"],"pubmed_authors":["Price-Kuehne F","D'Arco F","Crow YJ","Norrie J","Sudarsanam A","Maclean A","Bondet V","Duffy D","Murphy L","Kennel T","Donald A","Hemingway C","Reid KR","Wassmer E","Quigley AJ","El-Daher MT","Eleftheriou D","Livingstone K","Sutherland FJ","Ensor H","Munro AF","Rice GI","Battison C","Jack K","Forbes D","AGS‐RTIs Study Group","Parker RA","Moraitis E","Briggs TA","Shetty J","Mcmaster P","Hunt DP","Giddings A","Hong Y","Burleigh A","Parida A"],"additional_accession":[]},"is_claimable":false,"name":"Reverse transcriptase inhibitors in Aicardi-Goutieres syndrome: A crossover clinical trial.","description":"<h4>Aim</h4>To extend the findings of a previous clinical trial suggesting combined abacavir (ABC), lamivudine (3TC), and zidovudine (AZT) reduces type I interferon (IFN) signalling in Aicardi-Goutières syndrome (AGS).<h4>Method</h4>This was an open label, non-placebo-controlled phase II clinical trial (NCT04731103) in patients less than 16 years with any of five AGS genotypes. The effect of ABC or 3TC individually, or of combined ABC + 3TC + AZT, on IFN-stimulated gene (ISG) expression (primary outcome) and IFN-alpha protein (secondary outcome) in blood was assessed.<h4>Results</h4>Thirteen patients were recruited. Compliance was poor in the ABC + 3TC + AZT arm. No statistically significant effects were observed with ABC or 3TC, or with ABC + 3TC + AZT over 6 weeks. A statistically signif","dates":{"release":"2025-01-01T00:00:00Z","publication":"2025 Jun","modification":"2026-07-15T21:54:19.582Z","creation":"2025-04-04T01:54:38.927Z"},"accession":"S-EPMC7617231","cross_references":{"pubmed":["39630935"],"doi":["10.1111/dmcn.16199"]}}