Silencing mutant huntingtin by adeno-associated virus-mediated RNA interference ameliorates disease manifestations in the YAC128 mouse model of Huntington's disease (Q39795092)

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scientific article published on 21 March 2014

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English	Silencing mutant huntingtin by adeno-associated virus-mediated RNA interference ameliorates disease manifestations in the YAC128 mouse model of Huntington's disease	scientific article published on 21 March 2014

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scholarly article

1 reference

Europe PubMed Central

14 September 2017

Silencing mutant huntingtin by adeno-associated virus-mediated RNA interference ameliorates disease manifestations in the YAC128 mouse model of Huntington's disease (English)

1 reference

Europe PubMed Central

14 September 2017

Huntington's disease

0 references

RNA interference

1 reference

based on heuristic

inferred from title

author name string

Lisa M Stanek

1

1 reference

Europe PubMed Central

14 September 2017

Sergio P Sardi

2

1 reference

Europe PubMed Central

14 September 2017

Bryan Mastis

3

1 reference

Europe PubMed Central

14 September 2017

Amy R Richards

4

1 reference

Europe PubMed Central

14 September 2017

Christopher M Treleaven

5

1 reference

Europe PubMed Central

14 September 2017

Tatyana Taksir

6

1 reference

Europe PubMed Central

14 September 2017

Kuma Misra

7

1 reference

Europe PubMed Central

14 September 2017

Seng H Cheng

8

1 reference

Europe PubMed Central

14 September 2017

Lamya S Shihabuddin

9

1 reference

Europe PubMed Central

14 September 2017

publication date

21 March 2014

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Europe PubMed Central

14 September 2017

Human Gene Therapy

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Europe PubMed Central

14 September 2017

25

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Europe PubMed Central

14 September 2017

5

1 reference

Europe PubMed Central

14 September 2017

461-474

1 reference

Europe PubMed Central

14 September 2017

Long-term follow-up after gene therapy for canavan disease.

1 reference

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

1 reference

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22 September 2017

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

1 reference

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Long-term evaluation of a phase 1 study of AADC gene therapy for Parkinson's disease

1 reference

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22 September 2017

Allele-selective inhibition of trinucleotide repeat genes

1 reference

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22 September 2017

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

1 reference

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22 September 2017

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

1 reference

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

1 reference

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

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Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges

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AAV2-GAD gene therapy for advanced Parkinson's disease: a double-blind, sham-surgery controlled, randomised trial

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Oligonucleotide therapeutic approaches for Huntington disease

1 reference

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The widespread regulation of microRNA biogenesis, function and decay

1 reference

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Safety and tolerability of putaminal AADC gene therapy for Parkinson disease

1 reference

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22 September 2017

Sustained effects of nonallele-specific Huntingtin silencing

1 reference

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22 September 2017

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

1 reference

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Artificial microRNAs as siRNA shuttles: improved safety as compared to shRNAs in vitro and in vivo

1 reference

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

1 reference

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22 September 2017

Huntingtin modulates transcription, occupies gene promoters in vivo, and binds directly to DNA in a polyglutamine-dependent manner

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4028091

22 September 2017

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

1 reference

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22 September 2017

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

1 reference

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A century-old debate on protein aggregation and neurodegeneration enters the clinic

1 reference

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22 September 2017

Normal huntingtin function: an alternative approach to Huntington's disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4028091

22 September 2017

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

1 reference

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22 September 2017

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4028091

22 September 2017

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4028091

22 September 2017

Inclusion body formation reduces levels of mutant huntingtin and the risk of neuronal death

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4028091

22 September 2017

The functions of animal microRNAs

1 reference

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Cellular toxicity of polyglutamine expansion proteins: mechanism of transcription factor deactivation

1 reference

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22 September 2017

Wild-type huntingtin plays a role in brain development and neuronal survival

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4028091

22 September 2017

Huntingtin interacts with REST/NRSF to modulate the transcription of NRSE-controlled neuronal genes

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4028091

22 September 2017

Huntingtin aggregation and toxicity in Huntington's disease

1 reference

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Clinical protocol. Gene therapy of Canavan disease: AAV-2 vector for neurosurgical delivery of aspartoacylase gene (ASPA) to the human brain

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Widespread gene delivery and structure-specific patterns of expression in the brain after intraventricular injections of neonatal mice with an adeno-associated virus vector

1 reference

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Transcriptional dysregulation in Huntington's disease

1 reference

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22 September 2017

Reversal of neuropathology and motor dysfunction in a conditional model of Huntington's disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4028091

22 September 2017

Aggregation of huntingtin in neuronal intranuclear inclusions and dystrophic neurites in brain

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4028091

22 September 2017

Expression of normal and mutant huntingtin in the developing brain

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4028091

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Behavioral despair in mice: a primary screening test for antidepressants

1 reference

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22 September 2017

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4028091

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1 reference

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17 June 2018

Nuclear and neuropil aggregates in Huntington's disease: relationship to neuropathology.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4028091

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Neuropathological classification of Huntington's disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4028091

17 June 2018

Marked differences in neurochemistry and aggregates despite similar behavioural and neuropathological features of Huntington disease in the full-length BACHD and YAC128 mice

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4028091

20 August 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4028091

20 August 2018

Prevention of depressive behaviour in the YAC128 mouse model of Huntington disease by mutation at residue 586 of huntingtin

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4028091

20 August 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4028091

20 August 2018

Phenotypic abnormalities in the YAC128 mouse model of Huntington disease are penetrant on multiple genetic backgrounds and modulated by strain

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4028091

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Long-term clinical improvement in MPTP-lesioned primates after gene therapy with AAV-hAADC.

1 reference

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rAAV-mediated shRNA ameliorated neuropathology in Huntington disease model mouse

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4028091

20 August 2018

Selective deficits in the expression of striatal-enriched mRNAs in Huntington's disease.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4028091

20 August 2018

Selective degeneration and nuclear localization of mutant huntingtin in the YAC128 mouse model of Huntington disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4028091

20 August 2018

Decreased cAMP response element-mediated transcription: an early event in exon 1 and full-length cell models of Huntington's disease that contributes to polyglutamine pathogenesis

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4028091

20 August 2018

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4028091

20 August 2018

Regional and progressive thinning of the cortical ribbon in Huntington's disease

1 reference

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Noradrenergic lesions differentially alter the antidepressant-like effects of reboxetine in a modified forced swim test

1 reference

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20 August 2018

Huntingtin acts in the nucleus to induce apoptosis but death does not correlate with the formation of intranuclear inclusions.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4028091

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Huntingtin-encoded polyglutamine expansions form amyloid-like protein aggregates in vitro and in vivo

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4028091

20 August 2018

Dopamine D1 and D2 receptor gene expression in the striatum in Huntington's disease.

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4028091

20 August 2018

Reduced expression of preproenkephalin in striatal neurons from Huntington's disease patients

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4028091

20 August 2018

Singles engage the RNA interference pathway

1 reference

https://pubmed.ncbi.nlm.nih.gov/24484067

12 December 2020

based on heuristic

inferred from PubMed ID database lookup

Identifiers

10.1089/HUM.2013.200

1 reference

Europe PubMed Central

14 September 2017

1 reference

Europe PubMed Central

14 September 2017

1 reference

Europe PubMed Central

14 September 2017

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