RNA splicing modulation

RNA splicing modulation refers to the therapeutic targeting and alteration of the process by which precursor messenger RNA (pre-mRNA) is converted into mature mRNA through removal of introns and joining of exons. This process is catalyzed primarily by the spliceosome, a large ribonucleoprotein complex composed of small nuclear RNAs and proteins, as well as by self-splicing ribozymes in some cases. Modulating this process with small molecules or antisense oligonucleotides can alter gene expression patterns and has emerged as a promising strategy for treating cancers, genetic diseases, and infections. However, "RNA splicing modulation" itself is not a single molecular target but rather describes interventions on multiple components within the broader RNA processing machinery; thus it does not correspond to one canonical protein or receptor but rather to a class of mechanisms involving several molecular actors such as snRNPs, regulatory proteins, and catalytic RNAs involved in pre-mRNA processing.

Other names

Spliceosome modulationSplicing modulationModulation of RNA splicing

Inhibition or alteration of spliceosome activity by small molecules binding to conserved active sites in the spliceosome or self-splicing introns, thereby blocking or changing the normal sequence of splicing reactions and affecting gene expression outcomes.