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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Vydáno v:Mol Ther Nucleic Acids
Hlavní autoři: Sengupta, Kasturi, Mishra, Manoj K., Loro, Emanuele, Spencer, Melissa J., Pyle, April D., Khurana, Tejvir S.
Médium: Artigo
Jazyk:Inglês
Vydáno: American Society of Gene & Cell Therapy 2020
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Original Article
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC7554652/
https://ncbi.nlm.nih.gov/pubmed/33230452
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2020.08.031
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