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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...
Autores principales: | Sengupta, Kasturi, Mishra, Manoj K., Loro, Emanuele, Spencer, Melissa J., Pyle, April D., Khurana, Tejvir S. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
American Society of Gene & Cell Therapy
2020
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7554652/ https://www.ncbi.nlm.nih.gov/pubmed/33230452 http://dx.doi.org/10.1016/j.omtn.2020.08.031 |
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