Annual Meeting of the Japanese Society of Toxicology
The 50th Annual Meeting of the Japanese Society of Toxicology
Session ID : OS4-1
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Open Symposium 4: Discovery toxicology strategy for New modality
Considerations for evaluation of off-target effects of diversified modalities
*Takao INOUE
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CONFERENCE PROCEEDINGS FREE ACCESS

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Abstract

Off-target effects of gapmers and siRNAs are generally evaluated by methods to analyze the change in mRNA levels, such as microarray and quantitative PCR. On the other hand, the mechanism of action of oligonucleotide therapeutics is not limited to RNA cleavage but is becoming more diverse, including splicing control, miRNA control and RNA editing. In addition, the development of next-generation small molecule drugs that could compete or coexist with oligonucleotide therapeutics, such as PROTAC (TPD therapeutics) and RNA-targeting small molecule drugs, is progressing as related modalities. In principle, methods to analyze mRNA levels cannot be applied to the off-target evaluation of such advanced modalities, and it is necessary to consider evaluation methods based on the characteristics of the mechanism of action of each modality. In this talk, we would like to introduce our attempts of off-target evaluation for diversified modalities.

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© 2023 The Japanese Society of Toxicology
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