Genome Editing-Mediated Utrophin Upregulation in Duchenne Muscular Dystrophy Stem Cells

• Main Authors: Kasturi Sengupta (Author), Manoj K. Mishra (Author), Emanuele Loro (Author), Melissa J. Spencer (Author), April D. Pyle (Author), Tejvir S. Khurana (Author)
• Format: Book
• Published: Elsevier, 2020-12-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Utrophin upregulation is considered a promising therapeutic strategy for Duchenne muscular dystrophy (DMD). A number of microRNAs (miRNAs) post-transcriptionally regulate utrophin expression by binding their cognate sites in the 3' UTR. Previously we have shown that miRNA: UTRN repression can b...