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Ther Deliv. 2012 Sep;3(9):1061-76.

RNAi-based therapies for Huntington's disease: delivery challenges and opportunities.

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Department of Pharmaceutica Sciences, College of Pharmacy & Health Sciences, Butler University, Indianapolis, IN 46208, USA.


Huntington's disease (HD) is a polyglutamine neurodegenerative disease caused by a mutation in the HTT gene coding for the Huntingtin protein (HTT). Unfortunately, there is no cure for HD and there is also no known way to modify the disease progression. RNAi approaches offer the promise of a certain degree of control over the disease. However, there are several challenges in potential use of RNAi in the treatment of HD. This article will discuss the details of RNAi technology as applied to the treatment of HD, and novel approaches to overcome the drug delivery challenges.

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