HTT therapy refers to a range of therapeutic approaches aimed at treating Huntington's disease (HD), a neurodegenerative disorder caused by a mutation in the HTT gene. These therapies primarily focus on reducing the levels of the mutated huntingtin protein (mHTT), which is toxic to brain cells and causes the symptoms of HD.
How HTT Therapy Works
HTT therapies work by targeting the HTT gene or the mHTT protein itself. Some common approaches include:
- RNA interference (RNAi): This technique uses small interfering RNAs (siRNAs) to silence the expression of the HTT gene, thereby reducing the production of mHTT.
- Antisense oligonucleotides (ASOs): ASOs are short synthetic molecules that bind to the mRNA transcript of the HTT gene, preventing its translation into mHTT.
- Gene editing: This approach utilizes tools like CRISPR-Cas9 to directly modify the HTT gene, either by removing the mutated segment or correcting the mutation.
Benefits of HTT Therapy
HTT therapies hold significant promise for treating HD by:
- Reducing mHTT levels: By targeting the HTT gene or mHTT protein, these therapies aim to lower the accumulation of mHTT, potentially slowing down or preventing the progression of HD.
- Preserving brain function: Lowering mHTT levels may help protect brain cells from damage, potentially improving cognitive function, motor control, and overall quality of life for individuals with HD.
Examples of HTT Therapies in Development
Several HTT therapies are currently under development and undergoing clinical trials. Examples include:
- RG6042 (IONIS-HTTRx): An ASO designed to lower mHTT levels.
- BIIB090 (Tolebrutinib): An experimental drug targeting the huntingtin protein, potentially reducing its toxic effects.
- CRISPR-Cas9 gene editing therapies: Clinical trials are underway to evaluate the safety and efficacy of CRISPR-based therapies for HD.
Conclusion
HTT therapy is a rapidly evolving field, offering hope for a future where HD can be effectively managed and potentially even cured. While significant research and development are ongoing, these innovative therapies have the potential to transform the lives of people living with HD.
