Renadirsen, a Novel 2′OMeRNA/ENA<sup>®</sup> Chimera Antisense Oligonucleotide, Induces Robust Exon 45 Skipping for Dystrophin In Vivo

Renadirsen, a Novel 2′OMeRNA/ENA<sup>®</sup> Chimera Antisense Oligonucleotide, Induces Robust Exon 45 Skipping for Dystrophin In Vivo

Duchenne muscular dystrophy (DMD) is a progressive muscle-wasting disease caused by out-of-frame or nonsense mutation in the dystrophin gene. It begins with a loss of ambulation between 9 and 14 years of age, followed by various other symptoms including cardiac dysfunction. Exon skipping of patients...

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Bibliographic Details
Main Authors: Kentaro Ito, Hideo Takakusa, Masayo Kakuta, Akira Kanda, Nana Takagi, Hiroyuki Nagase, Nobuaki Watanabe, Daigo Asano, Ryoya Goda, Takeshi Masuda, Akifumi Nakamura, Yoshiyuki Onishi, Toshio Onoda, Makoto Koizumi, Yasuhiro Takeshima, Masafumi Matsuo, Kiyosumi Takaishi
Format: Article
Language:English
Published: MDPI AG 2021-09-01
Series:Current Issues in Molecular Biology
Subjects:
Duchenne muscular dystrophy
exon skipping
antisense oligonucleotide
dystrophin
2′-<i>O</i>,4′-<i>C</i>-ethylene-bridged nucleic acid (ENA)
renadirsen sodium
Online Access:https://www.mdpi.com/1467-3045/43/3/90

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https://www.mdpi.com/1467-3045/43/3/90

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