Issue 4, 2015
From the journal:

MedChemComm

Enhancement of exon skipping in mdx52 mice by 2′-O-methyl-2-thioribothymidine incorporation into phosphorothioate oligonucleotides†

Yoshiaki Masaki,a   Takeshi Inde,a   Tetsuya Nagata,b   Jun Tanihata,b   Takashi Kanamori,c   Kohji Seio,a   Shin'ichi Takedab  and  Mitsuo Sekine*a  

* Corresponding authors

a Department of Life Science, Tokyo Institute of Technology, J2-12, 4259 Nagatsuta-cho, Midoriku, Yokohama, Japan
E-mail: msekine@bio.titech.ac.jp

b Department of Molecular Therapy, Institute of Neuroscience, National Center of Neurology and Psychiatry, 4-1-1 Ogawa-Higashi, Kodaira, Tokyo 187-8502, Japan

c Education Academy of Computational Life Sciences, Tokyo Institute of Technology, 4259 Nagatsuta-cho, Midoriku, Yokohama, Japan

Abstract

Incorporation of 2′-O-methyl-2-thioribothymidine (s2Tm) into antisense oligoribonucleotides significantly enhanced the exon skipping activity in Duchenne muscular dystrophy model mice.

Graphical abstract: Enhancement of exon skipping in mdx52 mice by 2′-O-methyl-2-thioribothymidine incorporation into phosphorothioate oligonucleotides

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Article information

DOI
https://doi.org/10.1039/C4MD00468J
Article type
Concise Article
Submitted
15 Oct 2014
Accepted
15 Nov 2014
First published
08 Dec 2014

Med. Chem. Commun., 2015,6, 630-633

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Enhancement of exon skipping in mdx52 mice by 2′-O-methyl-2-thioribothymidine incorporation into phosphorothioate oligonucleotides

Y. Masaki, T. Inde, T. Nagata, J. Tanihata, T. Kanamori, K. Seio, S. Takeda and M. Sekine, Med. Chem. Commun., 2015, 6, 630 DOI: 10.1039/C4MD00468J

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