{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"submitter":["Zhang A"],"funding":["National Center for Medical Rehabilitation Research","NICHD NIH HHS","NIDDK NIH HHS","NCRR NIH HHS","National Institutes of Health","CTSI-CN"],"pagination":["838-849"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC4512206"],"repository":["biostudies-literature"],"omics_type":["Unknown"],"volume":["2"],"pubmed_abstract":["Phosphorodiamidate morpholino oligonucleotides (PMO) are used as a promising exon-skipping gene therapy for Duchenne Muscular Dystrophy (DMD). One potential complication of high dose PMO therapy is its transient accumulation in the kidneys. Therefore new urinary biomarkers are needed to monitor this treatment. Here, we carried out a pilot proteomic profiling study using stable isotope labeling in mammals (SILAM) strategy to identify new biomarkers to monitor the effect of PMO on the kidneys of the dystrophin deficient mouse model for DMD (mdx-23). We first assessed the baseline renal status of the mdx-23 mouse compared to the wild type (C57BL10) mouse, and then followed the renal outcome of mdx-23 mouse treated with a single high dose intravenous PMO injection (800 mg/kg). Surprisingly, un"],"journal":["Toxicology reports"],"pubmed_title":["The use of urinary and kidney SILAM proteomics to monitor kidney response to high dose morpholino oligonucleotides in the mdx mouse."],"pmcid":["PMC4512206"],"funding_grant_id":["R01 DK103564","R01 DK049419","U54 HD071601","UL1RR031988","R24 HD050846","U54HD07160","R01 DK-049419","UL1 RR031988","2R24HD050846-06"],"pubmed_authors":["Ray PE","Brown KJ","van den Anker JN","Hathout Y","Das JR","Rayavarapu S","Nagaraju K","Uaesoontrachoon K","Shaughnessy C","Hoffman EP","Zhang A"],"additional_accession":[]},"is_claimable":false,"name":"The use of urinary and kidney SILAM proteomics to monitor kidney response to high dose morpholino oligonucleotides in the mdx mouse.","description":"Phosphorodiamidate morpholino oligonucleotides (PMO) are used as a promising exon-skipping gene therapy for Duchenne Muscular Dystrophy (DMD). One potential complication of high dose PMO therapy is its transient accumulation in the kidneys. Therefore new urinary biomarkers are needed to monitor this treatment. Here, we carried out a pilot proteomic profiling study using stable isotope labeling in mammals (SILAM) strategy to identify new biomarkers to monitor the effect of PMO on the kidneys of the dystrophin deficient mouse model for DMD (mdx-23). We first assessed the baseline renal status of the mdx-23 mouse compared to the wild type (C57BL10) mouse, and then followed the renal outcome of mdx-23 mouse treated with a single high dose intravenous PMO injection (800 mg/kg). Surprisingly, un","dates":{"release":"2015-01-01T00:00:00Z","publication":"2015","modification":"2026-05-30T07:27:19.368Z","creation":"2019-03-27T01:55:42Z"},"accession":"S-EPMC4512206","cross_references":{"pubmed":["26213685"],"doi":["10.1016/j.toxrep.2015.05.008"]}}