نمایش پرونده ساده آیتم

dc.contributor.authorHu, RM
dc.contributor.authorBuck, NE
dc.contributor.authorKhaniani, MS
dc.contributor.authorWood, L
dc.contributor.authorWardan, H
dc.contributor.authorBenoist, JF
dc.contributor.authorLi, LL
dc.contributor.authorVadolas, J
dc.contributor.authorSarsero, JP
dc.contributor.authorIoannou, PA
dc.contributor.authorPeters, HL
dc.date.accessioned2018-08-26T08:15:38Z
dc.date.available2018-08-26T08:15:38Z
dc.date.issued2009
dc.identifier.urihttp://dspace.tbzmed.ac.ir:8080/xmlui/handle/123456789/51043
dc.description.abstractBackground Methylmalonic aciduria is an autosomal recessive inborn error of the propionate metabolic pathway. One form of this disorder is caused by mutations in methylmalonyl-coenzyme A mutase (MCM), resulting in reduced levels of enzyme activity. The pharmacological up-regulation of residual mutase activity is one approach to advance treatment strategies for individuals affected by this disorder. We describe the construction, characterization and use of a cellular genomic reporter assay for MCM expression that will potentially identify therapeutic pharmacological agents for methylmalonic aciduria treatment. Methods Homologous recombination was used to insert an enhanced green fluorescent protein (EGFP) cassette inframe before the last codon of exon 13 of the MCM gene (MUT) in a BAC clone. The construct was used to generate stable HeLa cell lines. EGFP expression was measured by flow cytometry and the real-time reverse transcriptase-polymerase chain reaction was used to quantify changes in MUT gene mRNA levels. Results The genomic reporter assay used to screen a selection of compounds. Cisplatin, zidovudine and adefovir were found to increase the levels of MCM mRNA and EGFP expression, providing support for the possible efficacy of these pharmacological compounds in treating methylmalonic aciduria. Conclusions This assay has the potential of being used in high-throughput screening of chemical libraries for the identification of novel compounds that specifically modulate the expression of MCM. Copyright (C) 2009 John Wiley Sons, Ltd.
dc.language.isoEnglish
dc.relation.ispartofJOURNAL OF GENE MEDICINE
dc.subjectgene induction
dc.subjectgenomic reporter assay
dc.subjectmethylmalonic aciduria
dc.titleGene induction for the treatment of methylmalonic aciduria
dc.typeArticle
dc.citation.volume11
dc.citation.issue4
dc.citation.spage361
dc.citation.epage369
dc.citation.indexWeb of science
dc.identifier.DOIhttps://doi.org/10.1002/jgm.1297


فایلهای درون آیتم

فایلهاسایزفرمتنمایش

هیچ فایل مرتبطی وجود ندارد

این آیتم در مجموعه های زیر مشاهده می شود

نمایش پرونده ساده آیتم