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Novel Disulfide-Bridged Bioresponsive Antisense Oligonucleotide Induces Efficient Splice Modulation in Muscle Myotubes in Vitro

[Image: see text] Splice-modulating antisense therapy has shown tremendous potential in therapeutic development in recent years with four FDA-approved antisense drugs since 2016. However, an efficient and nontoxic antisense oligonucleotide (AO) delivery system still remains as a major obstacle in nu...

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Detalles Bibliográficos
Autores principales: Le, Bao T., Kosbar, Tamer R., Veedu, Rakesh N.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Chemical Society 2020
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7391367/
https://www.ncbi.nlm.nih.gov/pubmed/32743177
http://dx.doi.org/10.1021/acsomega.0c01463