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Deletion of miR-146a enhances therapeutic protein restoration in model of dystrophin exon skipping

Duchenne muscular dystrophy (DMD) is a progressive muscle disease caused by the absence of dystrophin protein. One current DMD therapeutic strategy, exon skipping, produces a truncated dystrophin isoform using phosphorodiamidate morpholino oligomers (PMOs). However, the potential of exon skipping th...

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Detalles Bibliográficos
Autores principales:	McCormack, Nikki M., Calabrese, Kelsey A., Sun, Christina M., Tully, Christopher B., Heier, Christopher R., Fiorillo, Alyson A.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Cold Spring Harbor Laboratory 2023
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10197665/ https://www.ncbi.nlm.nih.gov/pubmed/37214870 http://dx.doi.org/10.1101/2023.05.09.540042

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