Advances in huntington disease drug discovery: novel approaches to model disease phenotypes (Q38160307)

30 August 2017

Advances in huntington disease drug discovery: novel approaches to model disease phenotypes (English)

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30 August 2017

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Jonathan Bard

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30 August 2017

Michael D Wall

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30 August 2017

Ovadia Lazari

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30 August 2017

Jamshid Arjomand

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30 August 2017

Ignacio Munoz-Sanjuan

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30 August 2017

6 November 2013

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Journal of Biomolecular Screening

30 August 2017

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30 August 2017

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30 August 2017

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30 August 2017

191-204

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

30 August 2017

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https://api.crossref.org/works/10.1177%2F1087057113510320

Motor abnormalities in premanifest persons with Huntington's disease: the PREDICT-HD study

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Psychiatric symptoms in Huntington's disease before diagnosis: the predict-HD study

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

The importance of integrating basic and clinical research toward the development of new therapies for Huntington disease

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

"Frontal" behaviors before the diagnosis of Huntington's disease and their relationship to markers of disease progression: evidence of early lack of awareness

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Molecular mechanisms and potential therapeutical targets in Huntington's disease

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

The corticostriatal pathway in Huntington's disease

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Pathological cell-cell interactions are necessary for striatal pathogenesis in a conditional mouse model of Huntington's disease

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Rodent genetic models of Huntington disease

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Transgenic rat model of Huntington's disease

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Expression profiling of Huntington's disease models suggests that brain-derived neurotrophic factor depletion plays a major role in striatal degeneration

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

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

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

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

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Replicating Huntington's disease phenotype in experimental animals

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Altered brain neurotransmitter receptors in transgenic mice expressing a portion of an abnormal human huntington disease gene

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Progressive alterations in the hypothalamic-pituitary-adrenal axis in the R6/2 transgenic mouse model of Huntington's disease

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Dopamine release is severely compromised in the R6/2 mouse model of Huntington's disease

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Decreased expression of striatal signaling genes in a mouse model of Huntington's disease

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Metabolic characterization of the R6/2 transgenic mouse model of Huntington's disease by high-resolution MAS 1H NMR spectroscopy

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

An ovine transgenic Huntington's disease model

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Identification and characterization of the miniature pig Huntington's disease gene homolog: evidence for conservation and polymorphism in the CAG triplet repeat

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Production of transgenic miniature pigs by pronuclear microinjection

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Executive decision-making in the domestic sheep

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

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

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Selective putaminal excitotoxic lesions in non-human primates model the movement disorder of Huntington disease.

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Striatal volume differences between non-human and human primates

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

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

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Formation of neuronal intranuclear inclusions underlies the neurological dysfunction in mice transgenic for the HD mutation

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

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

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Huntingtin aggregation and toxicity in Huntington's disease

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Huntington's disease: new paths to pathogenesis

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Protein aggregation and pathogenesis of Huntington's disease: mechanisms and correlations

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

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

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Oligoproline effects on polyglutamine conformation and aggregation

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Nucleation of huntingtin aggregation in cells

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

The aggregation-enhancing huntingtin N-terminus is helical in amyloid fibrils

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Slow amyloid nucleation via α-helix-rich oligomeric intermediates in short polyglutamine-containing huntingtin fragments.

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Inhibiting the nucleation of amyloid structure in a huntingtin fragment by targeting α-helix-rich oligomeric intermediates

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

CA150 expression delays striatal cell death in overexpression and knock-in conditions for mutant huntingtin neurotoxicity.

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Aggregated polyglutamine peptides delivered to nuclei are toxic to mammalian cells

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

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

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

The influence of huntingtin protein size on nuclear localization and cellular toxicity.

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

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

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

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

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Marked increase in mitochondrial DNA deletion levels in the cerebral cortex of Huntington's disease patients

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Reduction in enkephalin and substance P messenger RNA in the striatum of early grade Huntington's disease: a detailed cellular in situ hybridization study

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

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

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Altered neurotransmitter receptor expression in transgenic mouse models of Huntington's disease

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Decreased neuronal nitric oxide synthase messenger RNA and somatostatin messenger RNA in the striatum of Huntington's disease

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Transcriptional repression of PGC-1alpha by mutant huntingtin leads to mitochondrial dysfunction and neurodegeneration.

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Huntington's disease: revisiting the aggregation hypothesis in polyglutamine neurodegenerative diseases

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Cargo recognition failure is responsible for inefficient autophagy in Huntington's disease

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Clearance of mutant proteins as a therapeutic target in neurodegenerative diseases

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Autophagy gone awry in neurodegenerative diseases

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Novel targets for Huntington's disease in an mTOR-independent autophagy pathway

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

High-Throughput Multiplexed Quantitation of Protein Aggregation and Cytotoxicity in a Huntington's Disease Model

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

An aggregation sensing reporter identifies leflunomide and teriflunomide as polyglutamine aggregate inhibitors

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Genome-wide RNA interference screen identifies previously undescribed regulators of polyglutamine aggregation

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Modifiers of mutant huntingtin aggregation: functional conservation of C. elegans-modifiers of polyglutamine aggregation

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

RNAi screening in Drosophila cells identifies new modifiers of mutant huntingtin aggregation

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

A phenotypic screening assay for modulators of huntingtin-induced transcriptional dysregulation

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Discovery of a novel small-molecule targeting selective clearance of mutant huntingtin fragments.

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

A screen for enhancers of clearance identifies huntingtin as a heat shock protein 90 (Hsp90) client protein.

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Mutant huntingtin fragmentation in immune cells tracks Huntington's disease progression

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Huntington's disease genetics

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

A potent small molecule inhibits polyglutamine aggregation in Huntington's disease neurons and suppresses neurodegeneration in vivo

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

A small-molecule therapeutic lead for Huntington's disease: preclinical pharmacology and efficacy of C2-8 in the R6/2 transgenic mouse

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1087057113510320

3-(N-arylsulfamoyl)benzamides, inhibitors of human sirtuin type 2 (SIRT2)

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1087057113510320

Neuronal store-operated calcium entry pathway as a novel therapeutic target for Huntington's disease treatment

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1087057113510320

Mutant huntingtin alters MAPK signaling pathways in PC12 and striatal cells: ERK1/2 protects against mutant huntingtin-associated toxicity.

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1087057113510320

Dopamine D2 receptor stimulation potentiates PolyQ-Huntingtin-induced mouse striatal neuron dysfunctions via Rho/ROCK-II activation

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Combining comparative proteomics and molecular genetics uncovers regulators of synaptic and axonal stability and degeneration in vivo

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

A genome-scale RNA-interference screen identifies RRAS signaling as a pathologic feature of Huntington's disease

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

High-content chemical and RNAi screens for suppressors of neurotoxicity in a Huntington's disease model

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Large-scale functional RNAi screen in C. elegans identifies genes that regulate the dysfunction of mutant polyglutamine neurons.

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1087057113510320

Surface plasmon resonance characterization of specific binding of polyglutamine aggregation inhibitors to the expanded polyglutamine stretch

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Chaperone-like N-methyl peptide inhibitors of polyglutamine aggregation

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1087057113510320

Huntingtin localization in brains of normal and Huntington's disease patients

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1087057113510320

Composite primary neuronal high-content screening assay for Huntington's disease incorporating non-cell-autonomous interactions

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1087057113510320

High-throughput screening in primary neurons

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1087057113510320

Identification of anti-inflammatory targets for Huntington's disease using a brain slice-based screening assay

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1087057113510320

Characterization of neurophysiological and behavioral changes, MRI brain volumetry and 1H MRS in zQ175 knock-in mouse model of Huntington's disease.

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1087057113510320

Pathophysiology of Huntington's disease: time-dependent alterations in synaptic and receptor function.

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1087057113510320

Alterations in striatal synaptic transmission are consistent across genetic mouse models of Huntington's disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1087057113510320

Microelectrode arrays: a physiologically based neurotoxicity testing platform for the 21st century

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Lack of huntingtin promotes neural stem cells differentiation into glial cells while neurons expressing huntingtin with expanded polyglutamine tracts undergo cell death

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1087057113510320

Early transcriptional changes linked to naturally occurring Huntington's disease mutations in neural derivatives of human embryonic stem cells.

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1087057113510320

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1087057113510320

An evolutionary recent neuroepithelial cell adhesion function of huntingtin implicates ADAM10-Ncadherin

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Selective roles of normal and mutant huntingtin in neural induction and early neurogenesis

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Multiple phenotypes in Huntington disease mouse neural stem cells

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Derivation of human embryonic stem cell lines from embryos obtained after IVF and after PGD for monogenic disorders.

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Human embryonic stem cell models of Huntington disease.

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

A novel human embryonic stem cell-derived Huntington's disease neuronal model exhibits mutant huntingtin (mHTT) aggregates and soluble mHTT-dependent neurodegeneration

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Rhes, a striatal specific protein, mediates mutant-huntingtin cytotoxicity

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Mutant induced pluripotent stem cell lines recapitulate aspects of TDP-43 proteinopathies and reveal cell-specific vulnerability

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1087057113510320

Drug screening for ALS using patient-specific induced pluripotent stem cells

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Creation of an open-access, mutation-defined fibroblast resource for neurological disease research

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Disease-specific induced pluripotent stem cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1087057113510320

Induced pluripotent stem cells from patients with Huntington's disease show CAG-repeat-expansion-associated phenotypes

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1087057113510320

The first reported generation of several induced pluripotent stem cell lines from homozygous and heterozygous Huntington's disease patients demonstrates mutation related enhanced lysosomal activity

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Genetic correction of Huntington's disease phenotypes in induced pluripotent stem cells

21 January 2018

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https://api.crossref.org/works/10.1177%2F1087057113510320

Quantitative proteomic analysis of induced pluripotent stem cells derived from a human Huntington's disease patient

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1087057113510320

Neuronal properties, in vivo effects, and pathology of a Huntington's disease patient-derived induced pluripotent stem cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1087057113510320

Characterization of Human Huntington's Disease Cell Model from Induced Pluripotent Stem Cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1087057113510320

Striatal progenitors derived from human ES cells mature into DARPP32 neurons in vitro and in quinolinic acid-lesioned rats.

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1087057113510320

Human pluripotent stem cell differentiation into authentic striatal projection neurons

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1087057113510320

Astrocytes generated from patient induced pluripotent stem cells recapitulate features of Huntington's disease patient cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1087057113510320

Functional genomic screen of human stem cell differentiation reveals pathways involved in neurodevelopment and neurodegeneration.

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1087057113510320

Adapting human pluripotent stem cells to high-throughput and high-content screening

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1087057113510320

Small molecules enhance autophagy and reduce toxicity in Huntington's disease models.

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1087057113510320

Small molecule regulators of autophagy identified by an image-based high-throughput screen

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1087057113510320

Compounds blocking mutant huntingtin toxicity identified using a Huntington's disease neuronal cell model

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1087057113510320

Matrix metalloproteinases are modifiers of huntingtin proteolysis and toxicity in Huntington's disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1087057113510320

Spt4 is selectively required for transcription of extended trinucleotide repeats

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1087057113510320

Inhibition of polyglutamine protein aggregation and cell death by novel peptides identified by phage display screening

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1087057113510320

Pathogenic and Non-pathogenic Polyglutamine Tracts Have Similar Structural Properties: Towards a Length-dependent Toxicity Gradient

21 January 2018

1 reference

https://api.crossref.org/works/10.1177%2F1087057113510320

21 January 2018

Identifiers

DOI

10.1177/1087057113510320

1 reference

30 August 2017

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