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Pharmacokinetic/Pharmacodynamic Modeling of a Cell-Penetrating Peptide Phosphorodiamidate Morpholino Oligomer in mdx Mice

PURPOSE: Peptide-conjugated phosphorodiamidate morpholino oligomers (PPMOs) have shown promise in treating Duchenne muscular dystrophy (DMD). We evaluated a semi-mechanistic pharmacokinetic (PK) and pharmacodynamic (PD) model to capture the relationship between plasma and muscle tissue exposure/resp...

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Detalles Bibliográficos
Autores principales: Mukashyaka, Marie Claire, Wu, Chia-Ling, Ha, Kristin, Zhang, Jianbo, Wood, Jenna, Foley, Samantha, Mastis, Bryan, Jungels, Nino, Sun, Huadong, Shadid, Mohammad, Harriman, Shawn, Hadcock, John R.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer US 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8602220/
https://www.ncbi.nlm.nih.gov/pubmed/34671920
http://dx.doi.org/10.1007/s11095-021-03118-5

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