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[1AW-11-5] Therapeutic manipulation of IKBKAP mis-splicing with a small molecule to cure familial dysautonomia

Masahiko Ajiro1, Kei Iida1, Tomonari Awaya1, Krainer Adrian2, Masatoshi Hagiwara1 (1.Kyoto Univ. Grad. Sch. of Med., 2.Cold Spring Harbor Lab.)

l . Genetic Disorder(4 . Animal and Plant Physiology, and Diseases)

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