The Japanese Biochemical Society/The Molecular Biology Society of Japan

[2P-1437] Development of an orally available inhibitor of CLK1 for skipping a mutated dystrophin exon in Duchenne muscular dystrophy

〇Yukiya Sako1, Kensuke Ninomiya1, Yukiko Okuno2, Masayasu Toyomoto1, Atsushi Nishida3, Yuka Koike1, Kenji Ohe1, Isao Kii1, Suguru Yoshida5, Naohiro Hashimoto4, Takamitsu Hosoya5, Masafumi Matsuo3, Masatoshi Hagiwara1 (1.Dept. of Anat. and Deve. Bio., Grad. Sch. Med., Kyoto Univ., 2.Med. Res. Sprt. Cent., Kyoto Univ. Grad. Schl. Med., 3.Dept. of Med. Reha., Koubegakuin Univ., 4.Dept. of Reg. Med., Nat. Cent. Geriatr. and Gerontol., 5.IBB, Tokyo Medi. & Dent. Univ.)

muscular dystrophy、CLK1、splicing、SR protein、chemical compound

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