Modulation of Abnormal Splicing of RNA Diseases by Small Chemical Compounds
Pre-mRNA splicing is a critical step for protein gene expression in higher eukaryotes. Another mode of splicing, termed alternative splicing, contributes to diversity of the expressed proteins from the limited number of genes in chromosomes. Those steps are highly regulated and must be accurate. Therefore, disruption of splicing regulation often results in hereditary and sporadic diseases called as “RNA diseases” in human. Modulation of splicing by small chemical compounds and nucleic acids has been targeting aberrant splicing in those diseases. In this chapter, I will introduce several RNA diseases and splicing-target therapeutic approaches with chemical compounds. Unveiling molecular mechanism and correction of aberrant splicing by small chemical compounds are important for both RNA biologists and clinicians who desire therapies for those diseases.
KeywordsRNA splicing Chemical compound RNA disease Aberrant splicing
I would like to thank the people in Dr. Shin-Ichiro Takahashi lab (the University of Tokyo) for helpful discussion and comments. This work was supported by Grants-in-Aid for Scientific Research (Grant 23112706) from the Ministry of Education, Culture, Sports, Science and Technology (MEXT) of Japan. I apologize to all scientists whose research could not be properly discussed and cited in this review owing to space limitations.
Conflicts of Interest
The author declares no conflict of interest.
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