Progress and Challenges in RNA Interference Therapy for Huntington Disease
Scott Q. Harper, PhD
Arch Neurol. 2009;66(8):933-938.
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.
Author Affiliation: Department of Pediatrics, The Ohio State University Medical Center, and Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus.
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