What Therapeutic Approaches Target Mutant Huntingtin?
Several therapeutic strategies aim to mitigate the effects of mHTT. Gene silencing approaches, such as antisense oligonucleotides (ASOs) and RNA interference (RNAi), aim to reduce the expression of mutant huntingtin. Proteostasis modulators are being explored to enhance the degradation of mHTT aggregates. Additionally, small molecules and gene editing techniques like CRISPR-Cas9 hold promise for targeting the underlying genetic mutation.
