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

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

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 be all...

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Bibliographic Details
Main Authors: Kasturi Sengupta, Manoj K. Mishra, Emanuele Loro, Melissa J. Spencer, April D. Pyle, Tejvir S. Khurana
Format: Article
Language:English
Published: Elsevier 2020-12-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
utrophin
CRISPR-Cas9
IMTR
UTRNΔIMTR
DMD-hiPSC
Duchenne muscular dystrophy
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253120302602

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http://www.sciencedirect.com/science/article/pii/S2162253120302602

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