Sci Rep 2020 12 9;10(1):21492. Epub 2020 Dec 9.
Department of Physiology and Pennsylvania Muscle Institute, Perelman School of Medicine, University of Pennsylvania, 755 Clinical Research Building, Philadelphia, PA, 19104, USA.
Upregulation of utrophin, a dystrophin related protein, is considered a promising therapeutic approach for Duchenne muscular dystrophy (DMD). Utrophin expression is repressed at the post-transcriptional level by a set of miRNAs, among which let-7c is evolutionarily highly conserved. We designed PMO-based SBOs complementary to the let-7c binding site in UTRN 3'UTR, with the goal of inhibiting let-7c interaction with UTRN mRNA and thus upregulating utrophin. Read More