Gene therapy for Huntington's disease (HD) focuses on addressing the mutated HTT gene, responsible for producing toxic proteins that damage brain neurons. A prominent strategy under development involves gene silencing techniques, such as RNA interference (RNAi). This method delivers small RNA fragments that bind to the mutant HTT mRNA, effectively reducing the production of harmful proteins. Another approach utilizes viral vectors to introduce therapeutic DNA, aiming to modify or inhibit the mutated gene directly, which could potentially slow or halt the progression of Huntington's disease. While these therapies are still undergoing clinical trials, they show great promise in treating this debilitating neurodegenerative disorder.
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