Heteroduplex oligonucleotide technology boosts oligonucleotide splice switching activity of morpholino oligomers in a Duchenne muscular dystrophy mouse model

Heteroduplex oligonucleotide technology boosts oligonucleotide splice switching activity of morpholino oligomers in a Duchenne muscular dystrophy mouse model

Abstract The approval of splice-switching oligonucleotides with phosphorodiamidate morpholino oligomers (PMOs) for treating Duchenne muscular dystrophy (DMD) has advanced the field of oligonucleotide therapy. Despite this progress, PMOs encounter challenges such as poor tissue uptake, particularly i...

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Bibliographic Details
Main Authors: Juri Hasegawa, Tetsuya Nagata, Kensuke Ihara, Jun Tanihata, Satoe Ebihara, Kie Yoshida-Tanaka, Mitsugu Yanagidaira, Masahiro Ohara, Asuka Sasaki, Miyu Nakayama, Syunsuke Yamamoto, Takashi Ishii, Rintaro Iwata-Hara, Mitsuru Naito, Kanjiro Miyata, Fumika Sakaue, Takanori Yokota
Format: Article
Language:English
Published: Nature Portfolio 2024-09-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/s41467-024-48204-5

Internet

https://doi.org/10.1038/s41467-024-48204-5

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