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RNAi-based Therapies for Huntington‘s Disease: Delivery Challenges and Opportunities

, &
Pages 1061-1076 | Published online: 29 Aug 2012
 

Abstract

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.

Financial & competing interests disclosure

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

No writing assistance was utilized in the production of this manuscript.

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