MISC

査読有り
2012年6月

Challenges to congenital genetic disorders with "RNA-targeting" chemical compounds

PHARMACOLOGY & THERAPEUTICS
  • Yasushi Ogawa
  • ,
  • Masatoshi Hagiwara

134
3
開始ページ
298
終了ページ
305
記述言語
英語
掲載種別
書評論文,書評,文献紹介等
DOI
10.1016/j.pharmthera.2012.02.001
出版者・発行元
PERGAMON-ELSEVIER SCIENCE LTD

Patients of congenital diseases such as Down syndrome (DS) and Duchenne muscular dystrophy (DMD) have abnormalities in their chromosomes and/or genes. Therefore, it has been considered that drug treatments can serve to do little for these patients more than to patch over each symptom temporarily when it arises. Although we cannot normalize their chromosomes and genes with chemical drugs, we may be able to manipulate the amounts and patterns of mRNAs transcribed from patients' DNAs with small chemicals. Based on this simple idea, we have looked for chemical compounds which can be applicable for congenital diseases and found that protein kinase inhibitors such as INDY, TG003, and SRPIN340 are promising as clinical drugs for DS, DMD, and DDS, respectively. (C) 2012 Elsevier Inc. All rights reserved.

Web of Science ® 被引用回数 : 4

リンク情報
DOI
https://doi.org/10.1016/j.pharmthera.2012.02.001
PubMed
https://www.ncbi.nlm.nih.gov/pubmed/22342810
Web of Science
https://gateway.webofknowledge.com/gateway/Gateway.cgi?GWVersion=2&SrcAuth=JSTA_CEL&SrcApp=J_Gate_JST&DestLinkType=FullRecord&KeyUT=WOS:000303906200003&DestApp=WOS_CPL

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