MISC

査読有り
2015年4月

Modulation of Alternative Splicing with Chemical Compounds in New Therapeutics for Human Diseases

ACS CHEMICAL BIOLOGY
  • Kenji Ohe
  • ,
  • Masatoshi Hagiwara

10
4
開始ページ
914
終了ページ
924
記述言語
英語
掲載種別
書評論文,書評,文献紹介等
DOI
10.1021/cb500697f
出版者・発行元
AMER CHEMICAL SOC

Alternative splicing is a critical step where a limited number of human genes generate a complex and diverse proteome. Various diseases, including inherited diseases with abnormalities in the genome code, have been found to result in an aberrant mis-spliced transcript code with correlation to the resulting phenotype. Chemical compound-based and nucleic acid-based strategies are trying to target this mis-spliced transcript code. We will briefly mention about how to obtain splicing-modifying-compounds by high-throughput screening and overview of what is known about compounds that modify splicing pathways. The main focus will be on RNA-binding protein kinase inhibitors. In the main text, we will refer to diseases where splicing-modifying-compounds have been intensively investigated, with comparison to nucleic acid-based strategies. The information on their involvement in mis-splicing as well as nonsplicing events will be helpful in finding better compounds with less off-target effects for future implications in mis-splicing therapy.

Web of Science ® 被引用回数 : 20

リンク情報
DOI
https://doi.org/10.1021/cb500697f
PubMed
https://www.ncbi.nlm.nih.gov/pubmed/25560473
Web of Science
https://gateway.webofknowledge.com/gateway/Gateway.cgi?GWVersion=2&SrcAuth=JSTA_CEL&SrcApp=J_Gate_JST&DestLinkType=FullRecord&KeyUT=WOS:000353315100001&DestApp=WOS_CPL

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