Therapeutic manipulation of IKBKAP mis-splicing with a small molecule to cure familial dysautonomia.
Ajiro M, Awaya T, Kim YJ, Iida K, Denawa M, Tanaka N, Kurosawa R, Matsushima S, Shibata S, Sakamoto T, Studer L, Krainer AR, Hagiwara M.
Ajiro M, et al. Among authors: kim yj.
Nat Commun. 2021 Jul 23;12(1):4507. doi: 10.1038/s41467-021-24705-5.
Nat Commun. 2021.
PMID: 34301951
Free PMC article.