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Article

Progress and Challenges in RNA Interference Therapy for Huntington Disease

1 Aug 2009

Abstract excerpt

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...

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PMID: 19667213
DOI: 10.1001/archneurol.2009.180

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