Nonsense-mediated mRNA decay (NMD) UPF Inhibitors

UPF proteins are central components of the nonsense-mediated mRNA decay (NMD) pathway, a crucial post-transcriptional mechanism that regulates gene expression by degrading mRNA transcripts containing premature stop codons. This multiprotein complex, consisting of key members like UPF1, UPF2, and UPF3, plays a dual role in both mRNA nuclear export and surveillance of mRNA quality. UPF proteins identify and target aberrant mRNA transcripts for degradation, thereby the synthesis of potentially harmful truncated proteins. During splicing, these proteins interact with exon-junction complexes (EJCs), which are markers for newly synthesized mRNA, facilitating the recognition of mRNAs that should undergo NMD. The NMD pathway, regulated by UPF proteins, not only serves as a quality control mechanism but also plays a role in normal gene expression and the response to various cellular stresses.

Nonsense-mediated mRNA decay (NMD) UPF inhibitors are a class of chemical compounds designed to modulate the NMD pathway, a critical cellular mechanism responsible for the surveillance and degradation of mRNA transcripts containing premature termination codons (PTCs). This pathway, which is essential for maintaining the fidelity of gene expression, involves several key proteins, including UPF1, UPF2, and UPF3. NMD UPF inhibitors specifically target these proteins, disrupting their normal function and consequently affecting the NMD process. The development of these inhibitors stems from a deep understanding of the molecular biology of NMD, where UPF proteins are recognized as central players.