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Therapeutic manipulation of IKBKAP mis-splicing with a small molecule to cure familial dysautonomia.
Ajiro, Masahiko; Awaya, Tomonari; Kim, Young Jin; Iida, Kei; Denawa, Masatsugu; Tanaka, Nobuo; Kurosawa, Ryo; Matsushima, Shingo; Shibata, Saiko; Sakamoto, Tetsunori; Studer, Lorenz; Krainer, Adrian R; Hagiwara, Masatoshi.
Afiliação
  • Ajiro M; Department of Drug Discovery Medicine, Kyoto University Graduate School of Medicine, Kyoto, Japan.
  • Awaya T; Department of Anatomy and Developmental Biology, Kyoto University Graduate School of Medicine, Kyoto, Japan.
  • Kim YJ; Department of Anatomy and Developmental Biology, Kyoto University Graduate School of Medicine, Kyoto, Japan.
  • Iida K; Cold Spring Harbor Laboratory, Cold Spring Harbor, NY, USA.
  • Denawa M; Medical Research Support Center, Kyoto University Graduate School of Medicine, Kyoto, Japan.
  • Tanaka N; Medical Research Support Center, Kyoto University Graduate School of Medicine, Kyoto, Japan.
  • Kurosawa R; Medical Research Support Center, Kyoto University Graduate School of Medicine, Kyoto, Japan.
  • Matsushima S; Department of Anatomy and Developmental Biology, Kyoto University Graduate School of Medicine, Kyoto, Japan.
  • Shibata S; Department of Drug Discovery Medicine, Kyoto University Graduate School of Medicine, Kyoto, Japan.
  • Sakamoto T; Department of Anatomy and Developmental Biology, Kyoto University Graduate School of Medicine, Kyoto, Japan.
  • Studer L; Department of Drug Discovery Medicine, Kyoto University Graduate School of Medicine, Kyoto, Japan.
  • Krainer AR; Department of Anatomy and Developmental Biology, Kyoto University Graduate School of Medicine, Kyoto, Japan.
  • Hagiwara M; Department of Anatomy and Developmental Biology, Kyoto University Graduate School of Medicine, Kyoto, Japan.
Nat Commun ; 12(1): 4507, 2021 07 23.
Article em En | MEDLINE | ID: mdl-34301951
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Disautonomia Familiar / Processamento Alternativo / Fatores de Elongação da Transcrição / Mutação Tipo de estudo: Prognostic_studies Limite: Animals / Humans Idioma: En Ano de publicação: 2021 Tipo de documento: Article
Texto completo
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XML
PubMed Links
Buscar no Google

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Disautonomia Familiar / Processamento Alternativo / Fatores de Elongação da Transcrição / Mutação Tipo de estudo: Prognostic_studies Limite: Animals / Humans Idioma: En Ano de publicação: 2021 Tipo de documento: Article