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

• Egile Nagusiak: Kasturi Sengupta (Egilea), Manoj K. Mishra (Egilea), Emanuele Loro (Egilea), Melissa J. Spencer (Egilea), April D. Pyle (Egilea), Tejvir S. Khurana (Egilea)
• Formatua: Liburua
• Argitaratua: Elsevier, 2020-12-01T00:00:00Z.
• Gaiak: article
• Sarrera elektronikoa: 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...