(Q37327574)

English

Rational design of antisense oligonucleotides targeting single nucleotide polymorphisms for potent and allele selective suppression of mutant Huntingtin in the CNS.

scientific article published on 19 August 2013

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scholarly article

1 reference

Europe PubMed Central

PMC publication ID

19 August 2017

Rational design of antisense oligonucleotides targeting single nucleotide polymorphisms for potent and allele selective suppression of mutant Huntingtin in the CNS. (English)

1 reference

Europe PubMed Central

PMC publication ID

19 August 2017

single-nucleotide polymorphism

1 reference

based on heuristic

inferred from title

Michael R. Hayden

11

object named as

Michael R Hayden

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Europe PubMed Central

PMC publication ID

19 August 2017

author name string

Michael E Østergaard

1

1 reference

Europe PubMed Central

PMC publication ID

19 August 2017

Amber L Southwell

2

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Europe PubMed Central

PMC publication ID

19 August 2017

Holly Kordasiewicz

3

1 reference

Europe PubMed Central

PMC publication ID

19 August 2017

Andrew T Watt

4

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Europe PubMed Central

PMC publication ID

19 August 2017

Niels H Skotte

5

1 reference

Europe PubMed Central

PMC publication ID

19 August 2017

Crystal N Doty

6

1 reference

Europe PubMed Central

PMC publication ID

19 August 2017

Kuljeet Vaid

7

1 reference

Europe PubMed Central

PMC publication ID

19 August 2017

Erika B Villanueva

8

1 reference

Europe PubMed Central

PMC publication ID

19 August 2017

Eric E Swayze

9

1 reference

Europe PubMed Central

PMC publication ID

19 August 2017

C Frank Bennett

10

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Europe PubMed Central

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19 August 2017

Punit P Seth

12

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Europe PubMed Central

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19 August 2017

language of work or name

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publication date

19 August 2013

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Europe PubMed Central

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19 August 2017

Nucleic Acids Research

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Europe PubMed Central

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19 August 2017

41

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Europe PubMed Central

PMC publication ID

19 August 2017

21

1 reference

Europe PubMed Central

PMC publication ID

19 August 2017

9634-9650

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Europe PubMed Central

PMC publication ID

19 August 2017

copyright license

Creative Commons Attribution-NonCommercial 3.0 Unported

4 October 2016

1 reference

April 2022 Public Data File from Crossref

copyright status

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An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study

1 reference

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Aberrant splicing of HTT generates the pathogenic exon 1 protein in Huntington disease

1 reference

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A fully humanized transgenic mouse model of Huntington disease.

1 reference

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6 September 2017

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

1 reference

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

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Structure and nuclease resistance of 2′,4′-constrained 2′-O-methoxyethyl (cMOE) and 2′-O-ethyl (cEt) modified DNAs

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6 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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6 September 2017

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3834808

6 September 2017

Synthesis, Improved Antisense Activity and Structural Rationale for the Divergent RNA Affinities of 3′-Fluoro Hexitol Nucleic Acid (FHNA and Ara-FHNA) Modified Oligonucleotides

1 reference

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Targeting several CAG expansion diseases by a single antisense oligonucleotide

1 reference

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6 September 2017

Mechanisms of single-stranded phosphorothioate modified antisense oligonucleotide accumulation in hepatocytes

1 reference

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6 September 2017

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

1 reference

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6 September 2017

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

1 reference

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6 September 2017

Molecular Mechanisms and Potential Therapeutical Targets in Huntington's Disease

1 reference

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6 September 2017

RNA targeting therapeutics: molecular mechanisms of antisense oligonucleotides as a therapeutic platform

1 reference

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6 September 2017

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

1 reference

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6 September 2017

Structure of human RNase H1 complexed with an RNA/DNA hybrid: insight into HIV reverse transcription

1 reference

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

1 reference

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6 September 2017

Huntington's disease

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3834808

6 September 2017

Huntingtin inhibits caspase-3 activation

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3834808

6 September 2017

Human RNase H1 discriminates between subtle variations in the structure of the heteroduplex substrate

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3834808

6 September 2017

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

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3834808

6 September 2017

2'-Fluoroarabino- and arabinonucleic acid show different conformations, resulting in deviating RNA affinities and processing of their heteroduplexes with RNA by RNase H

1 reference

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Genetic medicines: treatment strategies for hereditary disorders

1 reference

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Diseases of unstable repeat expansion: mechanisms and common principles

1 reference

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6 September 2017

Structural requirements at the catalytic site of the heteroduplex substrate for human RNase H1 catalysis

1 reference

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6 September 2017

Determination of the role of the human RNase H1 in the pharmacology of DNA-like antisense drugs

1 reference

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Loss of huntingtin-mediated BDNF gene transcription in Huntington's disease

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The G x U wobble base pair. A fundamental building block of RNA structure crucial to RNA function in diverse biological systems

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Thermodynamics of RNA-RNA duplexes with 2- or 4-thiouridines: implications for antisense design and targeting a group I intron.

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Properties of cloned and expressed human RNase H1.

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Crystal structure and improved antisense properties of 2'-O-(2-methoxyethyl)-RNA

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

1 reference

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6 September 2017

Targeted disruption of the Huntington's disease gene results in embryonic lethality and behavioral and morphological changes in heterozygotes

1 reference

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6 September 2017

Increased apoptosis and early embryonic lethality in mice nullizygous for the Huntington's disease gene homologue

1 reference

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6 September 2017

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1 reference

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Short antisense oligonucleotides with novel 2'-4' conformationaly restricted nucleoside analogues show improved potency without increased toxicity in animals

1 reference

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14 September 2017

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

1 reference

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26 June 2018

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1 reference

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26 June 2018

Properties of 2'-fluorothymidine-containing oligonucleotides: interaction with restriction endonuclease EcoRV.

1 reference

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26 June 2018

Selective inhibition of mutant Ha-ras mRNA expression by antisense oligonucleotides

1 reference

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26 June 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=3834808

2 September 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=3834808

2 September 2018

Efficient reduction of target RNAs by small interfering RNA and RNase H-dependent antisense agents. A comparative analysis

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=3834808

2 September 2018

Long-range cooperativity due to C5-propynylation of oligopyrimidines enhances specific recognition by uridine of ribo-adenosine over ribo-guanosine

1 reference

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2 September 2018

Identifiers

10.1093/NAR/GKT725

1 reference

Europe PubMed Central

PMC publication ID

19 August 2017

PMC publication ID

1 reference

Europe PubMed Central

PMC publication ID

19 August 2017

PubMed publication ID

1 reference

Europe PubMed Central

PMC publication ID

19 August 2017

ResearchGate publication ID

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