siRNA p10A

siRNA p10A is an experimental allele-specific small interfering RNA (siRNA) designed for the treatment of Huntington's disease (HD). It functions by selectively silencing the mutant huntingtin (mHTT) mRNA while preserving the expression of the wild-type huntingtin (wtHTT) allele. The nomenclature "p10A" refers to the design of the siRNA guide strand, where a single nucleotide polymorphism (SNP) in the target HTT mRNA is matched at the 10th position of the siRNA guide strand with an Adenine. This specific positioning is optimized to maximize the discrimination between the mutant and normal alleles, leveraging the RNA interference (RNAi) pathway to degrade the toxic mutant transcript. Research into siRNA p10A and similar allele-specific siRNAs has demonstrated their ability to selectively suppress endogenous mutant huntingtin protein in patient-derived cells, offering a potential therapeutic strategy to mitigate the gain-of-function toxicity associated with HD without compromising the essential biological functions of the wild-type protein.

Other names

allele-specific siRNA p10A