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    Arch Neurol. 2009 Aug;66(8):933-8.

    Progress and challenges in RNA interference therapy for Huntington disease.

    Harper SQ.

    Department of Pediatrics, The Ohio State University Medical Center, and Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, 700 Children's Dr, Room WA2015, Columbus, OH 43205, USA. harper.104@osu.edu

    Erratum in:

    • Arch Neurol. 2009 Oct;66(10):1272.

    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.

    PMID: 19667213 [PubMed - indexed for MEDLINE]

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