siRNA-634 + siRNA-486

siRNA-634 + siRNA-486 is an experimental combination of two small interfering RNAs (siRNAs) designed to target the *Htt1a* transcript, a pathogenic splice variant of the Huntingtin (*HTT*) gene. In Huntington's disease, CAG repeat expansion leads to increased alternative processing of *HTT* mRNA into the *Htt1a* transcript, which encodes a highly aggregation-prone and toxic protein. This siRNA combination specifically targets and lowers *Htt1a* levels to delay the onset of molecular and neuropathological phenotypes, such as protein aggregation and transcriptional dysregulation. Developed by researchers associated with Atalanta Therapeutics, this approach represents a precision RNA interference strategy for treating Huntington's disease by focusing on toxic truncated protein products rather than, or in addition to, full-length Huntingtin.

Other names

Htt1a-targeting siRNAsHtt-1a-targeting siRNAsHtt 1a-targeting siRNAs