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Development of an exon skipping therapy for X-linked Alport syndrome with truncating variants in COL4A5.
Yamamura T, Horinouchi T, Adachi T, Terakawa M, Takaoka Y, Omachi K, Takasato M, Takaishi K, Shoji T, Onishi Y, Kanazawa Y, Koizumi M, Tomono Y, Sugano A, Shono A, Minamikawa S, Nagano C, Sakakibara N, Ishiko S, Aoto Y, Kamura M, Harita Y, Miura K, Kanda S, Morisada N, Rossanti R, Ye MJ, Nozu Y, Matsuo M, Kai H, Iijima K, Nozu K. Yamamura T, et al. Among authors: koizumi m. Nat Commun. 2020 Jun 2;11(1):2777. doi: 10.1038/s41467-020-16605-x. Nat Commun. 2020. PMID: 32488001 Free PMC article.
An improved synthesis of 2'-O,4'-C-ethylene nucleic acid (ENA) and thermodynamic studies of duplex formation containing the guanosine ENA unit.
Takagi-Sato M, Morita K, Onishi Y, Watahiki Y, Ishigaki T, Akita T, Tomita E, Kawakami J, Koizumi M. Takagi-Sato M, et al. Among authors: koizumi m. Nucleosides Nucleotides Nucleic Acids. 2020;39(6):838-852. doi: 10.1080/15257770.2019.1708389. Epub 2020 Jan 30. Nucleosides Nucleotides Nucleic Acids. 2020. PMID: 31997701
Renadirsen, a Novel 2'OMeRNA/ENA® Chimera Antisense Oligonucleotide, Induces Robust Exon 45 Skipping for Dystrophin In Vivo.
Ito K, Takakusa H, Kakuta M, Kanda A, Takagi N, Nagase H, Watanabe N, Asano D, Goda R, Masuda T, Nakamura A, Onishi Y, Onoda T, Koizumi M, Takeshima Y, Matsuo M, Takaishi K. Ito K, et al. Among authors: koizumi m. Curr Issues Mol Biol. 2021 Sep 25;43(3):1267-1281. doi: 10.3390/cimb43030090. Curr Issues Mol Biol. 2021. PMID: 34698059 Free PMC article.
Not all 2',4'-bridged modifications stabilize DNA/RNA duplexes.
Akita T, Tomita-Sudo E, Itoh S, Sakimoto N, Masuda T, Nakamura A, Onishi Y, Koizumi M, Kawakami J. Akita T, et al. Among authors: koizumi m. Nucleosides Nucleotides Nucleic Acids. 2024;43(1):57-64. doi: 10.1080/15257770.2023.2232414. Epub 2023 Jul 9. Nucleosides Nucleotides Nucleic Acids. 2024. PMID: 37424260
1,386 results