Investigating Synthetic Oligonucleotide Targeting of Mir31 in Duchenne Muscular Dystrophy.

ABSTRACT: Exon-skipping via synthetic antisense oligonucleotides represents one of the most promising potential therapies for Duchenne muscular dystrophy (DMD), yet this approach is highly sequence-specific and thus each oligonucleotide is of benefit to only a subset of patients. The discovery that dystrophin mRNA is subject to translational suppression by the microRNA miR31, and that miR31 is elevated in the muscle of DMD patients, raises the possibility that the same oligonucleotide chemistries employed for exon skipping could be directed toward relieving this translational block. This approach would act synergistically with exon skipping where possible, but by targeting the 3'UTR it would further be of benefit to the many DMD patients who express low levels of in-frame transcript. We here present

SUBMITTER: Hildyard JC 

PROVIDER: S-EPMC4972457 | biostudies-literature | 2016 Jun

REPOSITORIES: biostudies-literature