One of the most promising approaches to Huntington's Disease Treatmentinvolves gene silencing techniques to target and reduce the production of the toxic huntingtin protein. RNA interference (RNAi) and antisense oligonucleotides (ASOs) are two innovative methods employed for HTT-lowering therapies. RNAi utilizes small interfering RNA (siRNA) molecules to degrade the HTT messenger RNA (mRNA), preventing the synthesis of the toxic protein. ASOs, on the other hand, are short synthetic nucleic acid molecules designed to bind to the HTT mRNA, leading to its degradation. Both approaches have shown efficacy in preclinical studies and have progressed to clinical trials.
One notable breakthrough is the FDA approval of the ASO therapy called tominersen (previously known as RG6042 or IONIS-HTTRx). Clinical trials have demonstrated that tominersen effectively reduces HTT mRNA levels in the cerebrospinal fluid of HD patients. Although further research is necessary to establish its long-term safety and efficacy, this development provides an encouraging milestone in the treatment of HD.
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