RISC Inhibitors

RISC inhibitors, short for RNA-induced silencing complex inhibitors, belong to a class of molecules that play a pivotal role in the regulation of gene expression at the post-transcriptional level. These compounds primarily target the RNA interference (RNAi) pathway, a fundamental cellular mechanism that controls gene expression by degrading or suppressing the translation of specific messenger RNAs (mRNAs). The RNAi pathway is essential for maintaining the stability of the cellular transcriptome and for modulating various biological processes such as development, immunity, and genome defense.

RISC inhibitors work by interfering with the activity of the RNA-induced silencing complex (RISC), a multi-component molecular machinery that guides the sequence-specific recognition and degradation of target mRNAs. RISC consists of small RNA molecules, typically small interfering RNAs (siRNAs) or microRNAs (miRNAs), along with Argonaute proteins that act as the catalytic core of the complex. These small RNAs guide the RISC to complementary mRNA sequences, triggering either mRNA cleavage or translational repression, depending on the degree of complementarity. RISC inhibitors disrupt this process by binding to key components of the RISC complex or by preventing the loading of small RNAs onto Argonaute proteins, thereby impeding the silencing of specific target genes. Understanding the mechanisms of RISC inhibition can provide valuable insights into the regulation of gene expression and the development of tools for manipulating cellular processes that rely on RNA interference.