Ontology highlight
ABSTRACT:
SUBMITTER: Loro E
PROVIDER: S-EPMC7005813 | biostudies-literature | 2020 Feb
REPOSITORIES: biostudies-literature
Loro Emanuele E Sengupta Kasturi K Bogdanovich Sasha S Whig Kanupriya K Schultz David C DC Huryn Donna M DM Khurana Tejvir S TS
Scientific reports 20200207 1
Upregulation of endogenous utrophin offers great promise for treating DMD, as it can functionally compensate for the lack of dystrophin caused by DMD gene mutations, without the immunogenic concerns associated with delivering dystrophin. However, post-transcriptional repression mechanisms targeting the 5' and 3' untranslated regions (UTRs) of utrophin mRNA significantly limit the magnitude of utrophin upregulation achievable by promoter activation. Using a utrophin 5'3'UTR reporter assay, we per ...[more]