What are the Therapeutic Approaches Targeting Huntingtin Protein?

Several therapeutic strategies aim to target the huntingtin protein to treat Huntington’s disease. These include gene silencing approaches, such as antisense oligonucleotides and RNA interference, which reduce the production of mutant huntingtin. Other strategies focus on enhancing the clearance of mutant protein aggregates through autophagy or proteasome pathways. Histological studies of treated tissues help assess the efficacy of these therapeutic interventions by evaluating the reduction in mutant protein aggregates and the preservation of neuronal integrity.