siRNA 363125_C-16

siRNA 363125_C-16 is an experimental, allele-specific small interfering RNA (siRNA) designed for the treatment of Huntington's disease (HD). Developed by researchers at the University of Massachusetts Medical School, this molecule targets a specific single-nucleotide polymorphism (SNP), rs363125, located in the huntingtin (HTT) gene. By positioning the SNP-matching nucleotide at position 16 of the siRNA guide strand, the drug achieves high selectivity for the mutant HTT allele, enabling the degradation of mutant mRNA while sparing the wild-type mRNA. This approach aims to reduce the levels of toxic mutant huntingtin protein, which is the primary driver of neurodegeneration in HD, while maintaining the essential functions of the wild-type protein.

Other names

rs363125-targeted siRNArs-363125-targeted siRNArs 363125-targeted siRNAsiRNA 363125 C16siRNA363125 C16siRNA-363125 C16