siRNA p4G

siRNA p4G is an experimental allele-specific small interfering RNA (siRNA) designed to selectively silence the mutant huntingtin (mHTT) allele in patients with Huntington's disease. The designation "p4G" refers to the strategic placement of a mismatch—specifically a guanine (G) residue—at position 4 (p4) of the siRNA antisense strand relative to its 5' end. This design is engineered to exploit single nucleotide polymorphisms (SNPs) that are genetically linked to the Huntington's disease mutation, allowing the RNA-induced silencing complex (RISC) to preferentially recognize and degrade the mutant mRNA transcript while sparing the wild-type transcript. Developed primarily in academic research settings, such as the University of Massachusetts Medical School, siRNA p4G serves as a proof-of-concept for personalized RNA interference (RNAi) therapies that aim to reduce toxic protein levels without compromising the essential biological functions of the normal huntingtin protein.

Other names

p4G siRNAp-4G siRNAp 4G siRNAallele-specific siRNA p4G