Alternative splicing as a therapeutic target for human diseases

Kenneth J. Dery, Veronica Gusti, Shikha Gaur, John E. Shively, Yun Yen, Rajesh K. Gaur

研究成果: 書貢獻/報告類型章節

7 引文 斯高帕斯(Scopus)

摘要

The majority of eukaryotic genes undergo alternative splicing, an evolutionarily conserved phenomenon, to generate functionally diverse protein isoforms from a single transcript. The fact that defective pre-mRNA splicing can generate non-functional and often toxic proteins with catastrophic effects, accurate removal of introns and joining of exons is vital for cell homeostasis. Thus, molecular tools that could either silence a disease-causing gene or regulate its expression in trans will find many therapeutic applications. Here we present two RNA-based approaches, namely RNAi and theophylline-responsive riboswitch that can regulate gene expression by loss-of-function and modulation of splicing, respectively. These strategies are likely to continue to play an integral role in studying gene function and drug discovery.

原文英語
主出版物標題Therapeutic Applications of RNAi
主出版物子標題Methods and Protocols
編輯John Reidhaar-Olson, Cristina Rondinone
頁面127-144
頁數18
DOIs
出版狀態已發佈 - 十二月 1 2009
對外發佈Yes

出版系列

名字Methods in Molecular Biology
555
ISSN(列印)1064-3745

ASJC Scopus subject areas

  • Molecular Biology
  • Genetics

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