ASOs-a type of gene silencing treatment in which specially designed DNA molecules are used to switch off a gene.
The goal, for all these approaches, is to lower the amount of huntingtin proteinin brain cells. The huntingtin protein is the little machine made by cells as they follow the blueprint found in the HD gene. It’s the mutant huntingtin protein, not the mutation in the DNA, that most researchers believe causes the brain cell dysfunction that causes HD.
One quick reminder about how this process works is in order before we dive in. Cells follow instructions found in DNA, but they don’t use it directly to make a protein: they copy the instructions found in DNA into a sort of scratch copy of the genetic information, made from a closely related chemical called RNA. Scientists call this scratch copy messenger RNA or mRNA for short.
If you only remember one thing, remember this: a break in the chain anywhere will stop cells from making the huntingtin protein. In animal studies, lowering huntingtin protein led to big improvements in HD-like symptoms.
This approach to HD therapy is called huntingtin lowering and it’s behind the Ionis/Roche program. To read full article by Dr. Jeff Carroll: https://en.hdbuzz.net/266