siRNA p10G

siRNA p10G is an experimental, allele-specific small interfering RNA (siRNA) designed for the selective treatment of Huntington's disease (HD). It is engineered to silence the expression of the mutant huntingtin (mHTT) allele while preserving the expression of the wild-type allele by targeting single nucleotide polymorphisms (SNPs). The designation "p10G" refers to the placement of a Guanine (G) at the 10th position (p10) of the siRNA guide strand. This position is critical because it corresponds to the central catalytic cleavage site of the RNA-induced silencing complex (RISC), specifically where the Argonaute 2 (Ago2) protein cleaves the target mRNA. By placing the SNP-matching nucleotide at position 10, the siRNA achieves maximum discriminatory power between the mutant and wild-type sequences, leading to the degradation of mHTT mRNA and a subsequent reduction in toxic huntingtin protein levels. This research-stage molecule was primarily developed and characterized by researchers at the University of Massachusetts Medical School.

Other names

allele-specific siRNA p10Gp10 Guanine siRNAp-10 Guanine siRNAp 10 Guanine siRNA