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

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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Main Authors: Li Gan (Author), Leslie C.L. Wu (Author), Jenna A. Wood (Author), Monica Yao (Author), Chris M. Treleaven (Author), Nelsa L. Estrella (Author), Bruce M. Wentworth (Author), Gunnar J. Hanson (Author), Marco A. Passini (Author)
Format: Book
Published: Elsevier, 2022-12-01T00:00:00Z.
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