2009
DOI: 10.1001/archneurol.2009.180
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Progress and Challenges in RNA Interference Therapy for Huntington Disease

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Cited by 45 publications
(25 citation statements)
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“…One major approach currently under development in HD therapeutics is targeting HTT RNA for degradation using either antisense oligonucleotides or RNAi technologies. [40][41][42] Therapies targeting HTT RNA downstream of exon 1 will only reduce levels of a full-length HTT transcript leaving the exon 1 misspliced transcript untouched. We suggest an optimal strategy for HD therapeutics will target the RNA at the 5′ UTR or in exon 1 in order to reduce levels of both the full-length and the short mis-spliced exon 1 HTT transcript.…”
Section: Implications Of Mis-spliced Productmentioning
confidence: 99%
“…One major approach currently under development in HD therapeutics is targeting HTT RNA for degradation using either antisense oligonucleotides or RNAi technologies. [40][41][42] Therapies targeting HTT RNA downstream of exon 1 will only reduce levels of a full-length HTT transcript leaving the exon 1 misspliced transcript untouched. We suggest an optimal strategy for HD therapeutics will target the RNA at the 5′ UTR or in exon 1 in order to reduce levels of both the full-length and the short mis-spliced exon 1 HTT transcript.…”
Section: Implications Of Mis-spliced Productmentioning
confidence: 99%
“…In a series of RNAi applications in different HD transgenic rodent models, it was proven that silencing the mutant HD transgene can significantly inhibit neurodegeneration, improving motor control, and in some cases, it extended the lifespan of HD mice ( Figure 1). [2][3][4][5][6][7][8][9][10][11][12][13][14][15] More recently, strategies have been devised to specifically silence the mutant allele, whereas preserving expression of its wild-type counterpart. Because of the lack of a transgenic animal model expressing both alleles of human wild-type and mutant HD gene, allele-specific silencing is conducted in cells from HD patients currently.…”
Section: Introductionmentioning
confidence: 99%
“…As neurodegeneration proceeds, particularly quickly in transgenic mice expressing the 5¢-fragment of human HD protein (e.g., HD-N171-82Q, CAG140, R6/2, R6/1 and HD190qG), these animals were the first to be used for the proof-ofprinciple experiments with RNAi-based therapies ( Figure 1). [2][3][4] Most of these studies used RNAi targeted at a human HD transgene. If these RNAi eventually go to clinical trials, this interfering small RNA molecule will silence both the mutant and the wild-type huntingtin alleles.…”
Section: Introductionmentioning
confidence: 99%
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