(Q48243001)

9 February 2018

title

Artificial miRNAs Targeting Mutant Huntingtin Show Preferential Silencing In Vitro and In Vivo. (English)

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9 February 2018

author

Ryan L. Boudreau

3

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9 February 2018

author name string

Alex Mas Monteys

1

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9 February 2018

Matthew J Wilson

2

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9 February 2018

Ryan M Spengler

4

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9 February 2018

Beverly L Davidson

5

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9 February 2018

publication date

7 April 2015

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9 February 2018

Molecular Therapy. Nucleic acids

published in

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9 February 2018

volume

4

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9 February 2018

page(s)

e234

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9 February 2018

Trinucleotide repeat length instability and age of onset in Huntington's disease

cites work

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A worldwide study of the Huntington's disease mutation. The sensitivity and specificity of measuring CAG repeats

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Trinucleotide Repeat Disorders

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CAG repeat expansion in Huntington disease determines age at onset in a fully dominant fashion.

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Synthetic zinc finger repressors reduce mutant huntingtin expression in the brain of R6/2 mice

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Sustained therapeutic reversal of Huntington's disease by transient repression of huntingtin synthesis.

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RNA interference improves motor and neuropathological abnormalities in a Huntington's disease mouse model

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Sustained effects of nonallele-specific Huntingtin silencing

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Nonallele-specific silencing of mutant and wild-type huntingtin demonstrates therapeutic efficacy in Huntington's disease mice.

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Preclinical safety of RNAi-mediated HTT suppression in the rhesus macaque as a potential therapy for Huntington's disease

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Six-month partial suppression of Huntingtin is well tolerated in the adult rhesus striatum

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7 January 2021

Allele-specific silencing of mutant huntingtin and ataxin-3 genes by targeting expanded CAG repeats in mRNAs

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Allele-selective inhibition of huntingtin expression by switching to an miRNA-like RNAi mechanism

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Designing siRNA that distinguish between genes that differ by a single nucleotide

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Allele-specific suppression of mutant huntingtin using antisense oligonucleotides: providing a therapeutic option for all Huntington disease patients

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In vivo evaluation of candidate allele-specific mutant huntingtin gene silencing antisense oligonucleotides.

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Potent and selective antisense oligonucleotides targeting single-nucleotide polymorphisms in the Huntington disease gene / allele-specific silencing of mutant huntingtin

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Inhibition of mutant huntingtin expression by RNA duplex targeting expanded CAG repeats

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7 January 2021

Single-stranded RNAs use RNAi to potently and allele-selectively inhibit mutant huntingtin expression.

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Five siRNAs targeting three SNPs may provide therapy for three-quarters of Huntington's disease patients

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7 January 2021

Linking SNPs to CAG repeat length in Huntington's disease patients.

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Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: implications for the therapeutic development of RNAi

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Rational design of therapeutic siRNAs: minimizing off-targeting potential to improve the safety of RNAi therapy for Huntington's disease.

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Single nucleotide seed modification restores in vivo tolerability of a toxic artificial miRNA sequence in the mouse brain

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7 January 2021

Minimizing variables among hairpin-based RNAi vectors reveals the potency of shRNAs

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Structure of yeast Argonaute with guide RNA

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7 January 2021

Specificity of microRNA target selection in translational repression

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7 January 2021

Conserved seed pairing, often flanked by adenosines, indicates that thousands of human genes are microRNA targets

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7 January 2021

Full-length human mutant huntingtin with a stable polyglutamine repeat can elicit progressive and selective neuropathogenesis in BACHD mice

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7 January 2021

Generation of hairpin-based RNAi vectors for biological and therapeutic application.

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https://api.crossref.org/works/10.1038%2FMTNA.2015.7

7 January 2021

Identifiers

DOI

10.1038/MTNA.2015.7

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9 February 2018

PubMed ID

25849618

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9 February 2018