Progress and challenges in RNA interference therapy for Huntington disease.
نویسنده
چکیده
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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ورودعنوان ژورنال:
- Archives of neurology
دوره 66 8 شماره
صفحات -
تاریخ انتشار 2009