siRNA-10150

siRNA-10150 is a potent small interfering RNA (siRNA) designed to target and lower the levels of the Huntingtin (HTT) mRNA transcript. Developed as a research tool and potential therapeutic candidate for Huntington's disease (HD), it utilizes the RNA interference (RNAi) pathway to induce the degradation of HTT mRNA, thereby reducing the production of the huntingtin protein. In preclinical studies using mouse models such as zQ175, siRNA-10150 has been used to evaluate the efficacy of lowering full-length HTT transcripts compared to specific pathogenic variants like HTT1a. The compound has demonstrated significant potency and durability, particularly within the hippocampus. The development of this siRNA is associated with researchers from the University of Massachusetts Medical School and Atalanta Therapeutics.