Using non-coding small RNAs to develop therapies for Huntington's disease (Q37967034)

26 August 2017

title

Using non-coding small RNAs to develop therapies for Huntington's disease (English)

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26 August 2017

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Y Zhang

1

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26 August 2017

R M Friedlander

series ordinal

2

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26 August 2017

publication date

1 December 2011

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26 August 2017

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26 August 2017

volume

18

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26 August 2017

issue

12

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26 August 2017

page(s)

1139-1149

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A novel gene containing a trinucleotide repeat that is expanded and unstable on Huntington's disease chromosomes.

26 August 2017

cites work

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

Progress and challenges in RNA interference therapy for Huntington disease

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2011.170

RNAi: a potential therapy for the dominantly inherited nucleotide repeat diseases

21 January 2018

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RNAi therapy for neurodegenerative diseases.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

Clinico-pathological rescue of a model mouse of Huntington's disease by siRNA.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

AAV vector-mediated RNAi of mutant huntingtin expression is neuroprotective in a novel genetic rat model of Huntington's disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

Therapeutic silencing of mutant huntingtin with siRNA attenuates striatal and cortical neuropathology and behavioral deficits

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

Intrastriatal rAAV-mediated delivery of anti-huntingtin shRNAs induces partial reversal of disease progression in R6/1 Huntington's disease transgenic mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

Unexpected off-targeting effects of anti-huntingtin ribozymes and siRNA in vivo.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

rAAV-mediated shRNA ameliorated neuropathology in Huntington disease model mouse

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

Sustained effects of nonallele-specific Huntingtin silencing

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

High-capacity adenoviral vector-mediated reduction of huntingtin aggregate load in vitro and in vivo

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

Identification and allele-specific silencing of the mutant huntingtin allele in Huntington's disease patient-derived fibroblasts.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

Allele-specific silencing of mutant Huntington's disease gene

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

Allele-selective inhibition of mutant huntingtin by peptide nucleic acid-peptide conjugates, locked nucleic acid, and small interfering RNA.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

Expression of mutated huntingtin fragment in the putamen is sufficient to produce abnormal movement in non-human primates

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

A majority of Huntington's disease patients may be treatable by individualized allele-specific RNA interference.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

Mutant CAG repeats of Huntingtin transcript fold into hairpins, form nuclear foci and are targets for RNA interference

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

Allele-selective inhibition of mutant huntingtin expression with antisense oligonucleotides targeting the expanded CAG repeat

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

Intranuclear inclusions and neuritic aggregates in transgenic mice expressing a mutant N-terminal fragment of huntingtin

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

Exon 1 of the HD gene with an expanded CAG repeat is sufficient to cause a progressive neurological phenotype in transgenic mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

Decreased expression of hypothalamic neuropeptides in Huntington disease transgenic mice with expanded polyglutamine-EGFP fluorescent aggregates.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

Selective striatal neuronal loss in a YAC128 mouse model of Huntington disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

Progressive synaptic pathology of motor cortical neurons in a BAC transgenic mouse model of Huntington's disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

A YAC mouse model for Huntington's disease with full-length mutant huntingtin, cytoplasmic toxicity, and selective striatal neurodegeneration.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

CAG expansion in the Huntington disease gene is associated with a specific and targetable predisposing haplogroup

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

Structure and expression of the Huntington's disease gene: evidence against simple inactivation due to an expanded CAG repeat

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

Ancestral differences in the distribution of the delta 2642 glutamic acid polymorphism is associated with varying CAG repeat lengths on normal chromosomes: insights into the genetic evolution of Huntington disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

Haplotype analysis of the delta 2642 and (CAG)n polymorphisms in the Huntington's disease (HD) gene provides an explanation for an apparent 'founder' HD haplotype

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

Polymorphism analysis of the huntingtin gene in Italian families affected with Huntington disease.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

Genetic polymorphisms adjacent to the CAG repeat influence clinical features at onset in Huntington's disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

RNA structure of trinucleotide repeats associated with human neurological diseases

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

Trinucleotide repeats that expand in human disease form hairpin structures in vitro

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

Structural diversity of triplet repeat RNAs

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

Molecular architecture of CAG repeats in human disease related transcripts

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

Antisense inhibition of gene expression in cells by oligonucleotides incorporating locked nucleic acids: effect of mRNA target sequence and chimera design

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

Antisense PNA tridecamers targeted to the coding region of Ha-ras mRNA arrest polypeptide chain elongation

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

Expression profiling reveals off-target gene regulation by RNAi

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

A computational study of off-target effects of RNA interference

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

siRNA-mediated off-target gene silencing triggered by a 7 nt complementation

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

Widespread siRNA "off-target" transcript silencing mediated by seed region sequence complementarity

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

3' UTR seed matches, but not overall identity, are associated with RNAi off-targets

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

Specificity of microRNA target selection in translational repression

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

Kinetic analysis of the RNAi enzyme complex

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

Asymmetry in the assembly of the RNAi enzyme complex

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

Functional siRNAs and miRNAs Exhibit Strand Bias

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

Rational siRNA design for RNA interference

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

MicroRNA targeting specificity in mammals: determinants beyond seed pairing

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

Recombinant AAV viral vectors pseudotyped with viral capsids from serotypes 1, 2, and 5 display differential efficiency and cell tropism after delivery to different regions of the central nervous system

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

Transduction profiles of recombinant adeno-associated virus vectors derived from serotypes 2 and 5 in the nigrostriatal system of rats

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

Comparative analysis of adeno-associated viral vector serotypes 1, 2, 5, 7, and 8 in mouse brain

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

RNAi therapeutics: a potential new class of pharmaceutical drugs

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

Fully 2'-modified oligonucleotide duplexes with improved in vitro potency and stability compared to unmodified small interfering RNA.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

Synthesis and physical and physiological properties of 4'-thioRNA: application to post-modification of RNA aptamer toward NF-kappaB

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

Improving RNA interference in mammalian cells by 4'-thio-modified small interfering RNA (siRNA): effect on siRNA activity and nuclease stability when used in combination with 2'-O-alkyl modifications.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

Structural variations and stabilising modifications of synthetic siRNAs in mammalian cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

Position-specific chemical modification of siRNAs reduces "off-target" transcript silencing

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

Tolerance for mutations and chemical modifications in a siRNA.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

Stearylated octaarginine and artificial virus-like particles for transfection of siRNA into primary rat neurons

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

Stearylated arginine-rich peptides: a new class of transfection systems

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

A non-covalent peptide-based strategy for siRNA delivery.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

Aptamer mediated siRNA delivery

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

Transvascular delivery of small interfering RNA to the central nervous system.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

Sox11 transcription factor modulates peripheral nerve regeneration in adult mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

Highly efficient small interfering RNA delivery to primary mammalian neurons induces MicroRNA-like effects before mRNA degradation.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

Conjugate for efficient delivery of short interfering RNA (siRNA) into mammalian cells.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

Potential application of RNAi for understanding and therapy of neurodegenerative diseases

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

RNAi: a potential new class of therapeutic for human genetic disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

Intraneuronal aggregate formation and cell death after viral expression of expanded polyglutamine tracts in the adult rat brain.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

7 January 2021

Lentiviral-mediated delivery of mutant huntingtin in the striatum of rats induces a selective neuropathology modulated by polyglutamine repeat size, huntingtin expression levels, and protein length.

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

7 January 2021

Thermodynamic Stability of RNA Structures Formed by CNG Trinucleotide Repeats. Implication for Prediction of RNA Structure†

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

7 January 2021

Ribonuclease dicer cleaves triplet repeat hairpins into shorter repeats that silence specific targets

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

7 January 2021

CUG repeats present in myotonin kinase RNA form metastable "slippery" hairpins

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

7 January 2021

Recombinant adeno-associated virus type 2, 4, and 5 vectors: transduction of variant cell types and regions in the mammalian central nervous system.

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.170

7 January 2021

Identifiers

DOI

10.1038/GT.2011.170

1 reference

26 August 2017

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