Intrabody gene therapy ameliorates motor, cognitive, and neuropathological symptoms in multiple mouse models of Huntington's disease (Q30493159)

17 October 2017

Intrabody gene therapy ameliorates motor, cognitive, and neuropathological symptoms in multiple mouse models of Huntington's disease (English)

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17 October 2017

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Amber L Southwell

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17 October 2017

Jan Ko

series ordinal

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17 October 2017

Paul H Patterson

series ordinal

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17 October 2017

language of work or name

English

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publication date

1 October 2009

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17 October 2017

Journal of Neuroscience

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17 October 2017

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17 October 2017

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17 October 2017

13589-13602

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describes a project that uses

17 October 2017

ImageJ

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11 June 2022

https://www.ebi.ac.uk/europepmc/webservices/rest/PMC2822643/fullTextXML

based on heuristic

inferred from PubMed Central ID database lookup

cites work

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

20 June 2018

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

20 June 2018

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

20 June 2018

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Intranuclear degradation of polyglutamine aggregates by the ubiquitin-proteasome system.

20 June 2018

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Intrabodies binding the proline-rich domains of mutant huntingtin increase its turnover and reduce neurotoxicity

20 June 2018

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

20 June 2018

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Suppression of neuropil aggregates and neurological symptoms by an intracellular antibody implicates the cytoplasmic toxicity of mutant huntingtin

20 June 2018

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20 June 2018

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Soluble polyglutamine oligomers formed prior to inclusion body formation are cytotoxic

20 June 2018

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Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial

20 June 2018

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Isolation of a human single chain antibody fragment against oligomeric alpha-synuclein that inhibits aggregation and prevents alpha-synuclein-induced toxicity

20 June 2018

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Flanking sequences profoundly alter polyglutamine toxicity in yeast

20 June 2018

1 reference

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Absence of behavioral abnormalities and neurodegeneration in vivo despite widespread neuronal huntingtin inclusions

20 June 2018

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Suppression of Huntington's disease pathology in Drosophila by human single-chain Fv antibodies.

20 June 2018

1 reference

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A human single-chain Fv intrabody preferentially targets amino-terminal Huntingtin's fragments in striatal models of Huntington's disease

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2822643

beta-site specific intrabodies to decrease and prevent generation of Alzheimer's Abeta peptide.

20 June 2018

1 reference

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Potent inhibition of huntingtin aggregation and cytotoxicity by a disulfide bond-free single-domain intracellular antibody

20 June 2018

1 reference

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A human single-chain Fv intrabody blocks aberrant cellular effects of overexpressed alpha-synuclein

20 June 2018

1 reference

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

20 June 2018

1 reference

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Recombinant AAV-mediated gene delivery to the central nervous system.

20 June 2018

1 reference

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Common structure of soluble amyloid oligomers implies common mechanism of pathogenesis

20 June 2018

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Genetic fate of recombinant adeno-associated virus vector genomes in muscle

20 June 2018

1 reference

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Dose and promoter effects of adeno-associated viral vector for green fluorescent protein expression in the rat brain

20 June 2018

1 reference

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Hippocampal damage and exploratory preferences in rats: memory for objects, places, and contexts

20 June 2018

1 reference

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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.

20 June 2018

1 reference

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Inherent toxicity of aggregates implies a common mechanism for protein misfolding diseases

20 June 2018

1 reference

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Lessons from animal models of Huntington's disease

20 June 2018

1 reference

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Effects of intracellular expression of anti-huntingtin antibodies of various specificities on mutant huntingtin aggregation and toxicity

20 June 2018

1 reference

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New anti-huntingtin monoclonal antibodies: implications for huntingtin conformation and its binding proteins

20 June 2018

1 reference

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Human single-chain Fv intrabodies counteract in situ huntingtin aggregation in cellular models of Huntington's disease

20 June 2018

1 reference

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Tropism of AAV-2 vectors for neurons of the globus pallidus

20 June 2018

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Adeno-associated virus vectors: activity and applications in the CNS.

20 June 2018

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Immune responses to adenovirus and adeno-associated virus in humans.

20 June 2018

1 reference

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Evidence for both the nucleus and cytoplasm as subcellular sites of pathogenesis in Huntington's disease in cell culture and in transgenic mice expressing mutant huntingtin.

20 June 2018

1 reference

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Selective and rapid uptake of adeno-associated virus type 2 in brain

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2822643

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

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2822643

A stable human-derived packaging cell line for production of high titer retrovirus/vesicular stomatitis virus G pseudotypes

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2822643

Differential and persistent expression patterns of CNS gene transfer by an adeno-associated virus (AAV) vector

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2822643

In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2822643

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

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2822643

FVB/N: an inbred mouse strain preferable for transgenic analyses

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2822643

Polyglutamine disruption of the huntingtin exon 1 N terminus triggers a complex aggregation mechanism

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2822643

Safety and tolerability of intraputaminal delivery of CERE-120 (adeno-associated virus serotype 2-neurturin) to patients with idiopathic Parkinson's disease: an open-label, phase I trial

26 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2822643

Combinational approach of intrabody with enhanced Hsp70 expression addresses multiple pathologies in a fly model of Huntington's disease.

26 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2822643

Perirhinal cortex damage and anterograde object-recognition in rats after long retention intervals.

26 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2822643

Production and purification of lentiviral vectors

26 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2822643

Cytosolic chaperonin prevents polyglutamine toxicity with altering the aggregation state.

26 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2822643

Chaperonin TRiC promotes the assembly of polyQ expansion proteins into nontoxic oligomers

26 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2822643

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

26 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2822643

Cleavage at the caspase-6 site is required for neuronal dysfunction and degeneration due to mutant huntingtin

26 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2822643

Immune responses to AAV in a phase I study for Canavan disease

26 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2822643

Chronology of behavioral symptoms and neuropathological sequela in R6/2 Huntington's disease transgenic mice

26 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2822643

Cognitive dysfunction precedes neuropathology and motor abnormalities in the YAC128 mouse model of Huntington's disease.

26 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2822643

Nuclear-targeting of mutant huntingtin fragments produces Huntington's disease-like phenotypes in transgenic mice

26 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2822643

A single-chain Fv intrabody provides functional protection against the effects of mutant protein in an organotypic slice culture model of Huntington's disease

26 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2822643

Early and reversible neuropathology induced by tetracycline-regulated lentiviral overexpression of mutant huntingtin in rat striatum

26 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2822643

Recombinant AAV serotype 1 transduction efficiency and tropism in the murine brain

26 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2822643

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

26 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2822643

Pivotal role of oligomerization in expanded polyglutamine neurodegenerative disorders.

26 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2822643

Exchange of surface proteins impacts on viral vector cellular specificity and transduction characteristics: the retina as a model

26 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2822643

Nuclear targeting of mutant Huntingtin increases toxicity

26 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2822643

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

26 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2822643

Neuron-specific transduction in the rat septohippocampal or nigrostriatal pathway by recombinant adeno-associated virus vectors

26 November 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2822643

10.1523/JNEUROSCI.4286-09.2009

26 November 2018

Identifiers

DOI

1 reference

17 October 2017

1 reference

17 October 2017

PubMed publication ID

19864571

1 reference