RNAi藥物
RNAi technology refers to highly conserved, double-stranded RNA induced, highly specific degradation of homologous mrnas in the evolutionary process, and is a post-transcriptional gene silencing (PTGS) that degrades mrnas.
The main mechanism of RNAi is that long double RNA is cleaved into short RNA and binds with protein to form siRNA-induced interference complex (RISC). After the degradation of the righteous strand of short RNA, RISC binds to specific mRNA to degrade the mRNA and eventually silences the expression of specific genes.
Known RNAi mainly include siRNA, miRNA and piRNA.
ASO藥物
ASO refers to a single strand of DNA or RNA sequence that is complementary to the mRNA of the target gene, usually consisting of a dozen to dozens of bases and produced by chemical synthesis.
After some specific chemical modifications of ASO, ASO drugs enter cells in a certain way and can specifically regulate the expression of target genes.
RNAi藥物、ASO藥物的作用機制
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