Final answer:
ASO targets for treating HD can include mutant huntingtin protein or huntingtin mRNA. ASOs can be designed to bind to these targets and interfere with their function, potentially slowing down the progression of HD. ASOs can also target other genes or proteins involved in the disease pathway.
Step-by-step explanation:
To treat HD, ASO targets can include genes or proteins involved in the disease process. ASOs can be designed to specifically bind to the target and interfere with its function. Some potential targets for ASO therapy in HD include mutant huntingtin protein (mHTT) or huntingtin mRNA.
By targeting mHTT, ASOs can reduce the production of this abnormal protein and potentially slow down the progression of HD. ASOs can also target huntingtin mRNA to prevent the translation of the mRNA into mHTT. This approach aims to decrease the levels of mHTT in the cells.
Furthermore, ASOs can be used to target other genes or proteins that are implicated in the disease pathway to modulate their expression or function. This can include genes involved in oxidative stress, inflammation, or neuronal survival.