A cell-penetrating peptide enhances delivery and efficacy of phosphorodiamidate morpholino oligomers in mdx mice

Antisense RNA technology is a strategy for the treatment of Duchenne muscular dystrophy (DMD), a progressive and universally fatal X-linked neuromuscular disease caused by frameshift mutations in the gene encoding dystrophin. Phosphorodiamidate morpholino oligomers (PMOs) are an antisense RNA platfo...

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Detalles Bibliográficos
Autores principales: Gan, Li, Wu, Leslie C.L., Wood, Jenna A., Yao, Monica, Treleaven, Chris M., Estrella, Nelsa L., Wentworth, Bruce M., Hanson, Gunnar J., Passini, Marco A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2022
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9483789/
https://www.ncbi.nlm.nih.gov/pubmed/36189424
http://dx.doi.org/10.1016/j.omtn.2022.08.019

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9483789/
https://www.ncbi.nlm.nih.gov/pubmed/36189424
http://dx.doi.org/10.1016/j.omtn.2022.08.019

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