Abstract
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.
Publication types
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Research Support, Non-U.S. Gov't
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Review
MeSH terms
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Animals
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Base Pairing / genetics
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Disease Models, Animal
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Gene Silencing*
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Genes, Dominant / genetics
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Genetic Therapy*
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Genetic Vectors / genetics
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Genetic Vectors / therapeutic use
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Humans
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Huntingtin Protein
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Huntington Disease / genetics*
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Huntington Disease / therapy*
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Mice
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MicroRNAs / genetics
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Nerve Tissue Proteins / genetics*
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Nuclear Proteins / genetics*
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Prospective Studies
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RNA Interference / physiology*
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RNA, Messenger / genetics
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RNA-Induced Silencing Complex / genetics
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Transfection / methods
Substances
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HTT protein, human
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Huntingtin Protein
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MicroRNAs
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Nerve Tissue Proteins
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Nuclear Proteins
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RNA, Messenger
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RNA-Induced Silencing Complex