Final answer:
ASO to treat HD involves using RNA-targeting small molecules to reduce the levels of the mutant huntingtin protein in cells.
Step-by-step explanation:
[ASO](Advanced Splicing Optimization) to treat HD (Huntington's Disease) involves using RNA-targeting small molecules, also known as ASOs, to modulate gene expression and reduce the levels of the mutant huntingtin protein (mHTT) in cells.
The ASOs work by binding to the messenger RNA (mRNA) that carries the instructions for producing mHTT and promoting its degradation.
This approach aims to slow down or halt the progression of HD by reducing the accumulation of toxic mHTT in affected cells.
Learn more about ASO treatment for HD