Huntington's Disease Clinical Research - Reducing Huntingtin Production

Reducing Huntingtin Production

Gene silencing aims to reduce the production of the mutant protein, since HD is caused by a single dominant gene encoding a toxic protein. Gene silencing experiments in mouse models have shown that when the expression of mHtt is reduced, symptoms improve. Safety of non-allele specific RNAi and ASO gene silencing has now been demonstrated in mice and the large, human-like brains of primates. but clinical delivery of RNAi therapy remains problematic. Allele-specific silencing attempts to silence mutant Htt while leaving wild-type Htt untouched. One way of accomplishing this is to identify polymorphisms present on only one allele and produce gene silencing drugs that target polymorphisms in only the mutant allele.