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Development of DG9 peptide-conjugated single- and multi-exon skipping therapies for the treatment of Duchenne muscular dystrophy by Kenji Rowel Q. Lim; Stanley Woo; Dyanna Melo; Yiqing Huang; Kasia Dzierlega; Md Nur Ahad Shah; Tejal Aslesh; Rohini Roy Roshmi; Yusuke Echigoya; Rika Maruyama; Hong M. Moulton; Toshifumi Yokota is a Biochemistry, Genetics and Molecular Biology article available to read on EtoBox.

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## Significance Duchenne muscular dystrophy (DMD) is a fatal disorder of progressive body-wide muscle weakness, considered the most common muscular dystrophy worldwide. Most patients have out-of-frame deletions in the __DMD__ gene, leading to dystrophin absence in muscle. There is no cure for DMD, but exon skipping is emerging as a potential therapy that uses antisense oligonucleotides to convert out-of-frame to in-frame mutations, enabling the production of truncated, partially functional dystrophin. Currently approved exon skipping therapies, however, have limited applicability and efficacy. Here, we developed a more economical approach to skip __DMD__ exons 45 to 55 (a strategy that could treat nearly half of all DMD patients) and identified DG9 peptide conjugation as a powerful way to improve exon skipping efficiencies in vivo.

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Author

Kenji Rowel Q. Lim; Stanley Woo; Dyanna Melo; Yiqing Huang; Kasia Dzierlega; Md Nur Ahad Shah; Tejal Aslesh; Rohini Roy Roshmi; Yusuke Echigoya; Rika Maruyama; Hong M. Moulton; Toshifumi Yokota

Publisher

Proceedings of the National Academy of Sciences

Published

2022

Language

EN

Field

Biochemistry, Genetics and Molecular Biology (Life Sciences)