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RNA interference is being developed as a prospective therapy to silence mutant huntingtin expression in Huntington disease, though important challenges remain.
This review discusses the current progress and remaining challenges of developing RNA interference therapy to silence mutant huntingtin expression in Huntington disease.
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Investigational RNAi for Huntington disease requires further validation; leaves open translation from models to patients.
Huntington disease is an incurable, dominant neurodegenerative disorder caused by polyglutamine repeat expansion in the huntingtin protein. Reducing mutant huntingtin expression may offer a treatment for Huntington disease. RNA interference has emerged as a powerful method to silence dominant disease genes. As such, it is being developed as a prospective Huntington disease therapy. Here I discuss the current progress and important remaining challenges of RNA interference therapy for Huntington disease.
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Scott Q. Harper (2009) conducted a review in Huntington disease. RNA interference therapy was evaluated. RNA interference is being developed as a prospective therapy to silence mutant huntingtin expression in Huntington disease, though important challenges remain.
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