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RAN Translation in Huntington Disease

Neuron (Cambridge, Mass.), 2015-11, Vol.88 (4), p.667-677

2015

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Use of single guided Cas9 nickase to facilitate precise and efficient genome editing in human iPSCs

Scientific reports, 2021-05, Vol.11 (1), p.9865-9865, Article 9865

2021

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Synaptic dysregulation in a human iPS cell model of mental disorders

Nature (London), 2014-11, Vol.515 (7527), p.414-418

2014

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Huntington disease: natural history, biomarkers and prospects for therapeutics

Nature reviews. Neurology, 2014-04, Vol.10 (4), p.204-216

2014

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An Antisense CAG Repeat Transcript at JPH3 Locus Mediates Expanded Polyglutamine Protein Toxicity in Huntington's Disease-like 2 Mice

Neuron (Cambridge, Mass.), 2011-05, Vol.70 (3), p.427-440

2011

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

Molecular brain, 2012-05, Vol.5 (1), p.17-17, Article 17

2012

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Prevalence of Psychotic Symptoms in a Community-Based Parkinson Disease Sample

The American journal of geriatric psychiatry, 2012-02, Vol.20 (2), p.123-132

2012

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Generation of a human induced pluripotent stem cell line JHUi004-A with heterozygous mutation for spinocerebellar ataxia type 12 using genome editing

Stem cell research, 2024-06, Vol.77, p.103441, Article 103441

2024

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Age, CAG repeat length, and clinical progression in Huntington's disease

Movement disorders, 2012-02, Vol.27 (2), p.272-276

2012

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A high-throughput screening to identify small molecules that suppress huntingtin promoter activity or activate huntingtin-antisense promoter activity

Scientific reports, 2021-03, Vol.11 (1), p.6157-13, Article 6157

2021

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Generation of a human induced pluripotent stem cell line JHUi003-A with homozygous mutation for spinocerebellar ataxia type 12 using genome editing

Stem cell research, 2021-05, Vol.53, p.102346-102346, Article 102346

2021

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Pharmacological rescue in patient iPSC and mouse models with a rare DISC1 mutation

Nature communications, 2021-03, Vol.12 (1), p.1398-11, Article 1398

2021

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Loss of junctophilin-3 contributes to huntington disease-like 2 pathogenesis

Annals of neurology, 2012-02, Vol.71 (2), p.245-257

2012

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Author Correction: Pharmacological rescue in patient iPSC and mouse models with a rare DISC1 mutation

Nature communications, 2021-04, Vol.12 (1), p.2570-2570, Article 2570

2021

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Phosphorodiamidate morpholino oligomers suppress mutant huntingtin expression and attenuate neurotoxicity

Human molecular genetics, 2014-12, Vol.23 (23), p.6302-6317

2014

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Analysis of differential gene expression mediated by clozapine in human postmortem brains

Schizophrenia research, 2017-07, Vol.185, p.58-66

2017

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Emerging differences between Huntington's disease-like 2 and Huntington's disease: A comparison using MRI brain volumetry

NeuroImage clinical, 2019-01, Vol.21, p.101666, Article 101666

2019

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A Systematic Review of the Huntington Disease-Like 2 Phenotype

Journal of Huntington's disease, 2017-01, Vol.6 (1), p.37-46

2017

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Differential Changes in Functional Connectivity of Striatum-Prefrontal and Striatum-Motor Circuits in Premanifest Huntington’s Disease

Neuro-degenerative diseases, 2019-11, Vol.19 (2), p.78-87

2019

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Pharmacologic Treatment of Anxiety Disorders in Parkinson Disease

The American journal of geriatric psychiatry, 2013-06, Vol.21 (6), p.520-528

2013

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Research Domain Criteria: Strengths, Weaknesses, and Potential Alternatives for Future Psychiatric Research

Complex psychiatry, 2019-08, Vol.5 (4), p.218-236

2019

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The timing and severity of clozapine-associated neutropenia in the US: Is the risk overstated?

Schizophrenia research, 2024-10, Vol.272, p.104-109

2024

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Role of Bβ1 overexpression in the pathogenesis of SCA12

Movement disorders, 2024-05

2024

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Bidirectional Transcription at the PPP2R2B Gene Locus in Spinocerebellar Ataxia Type 12

Movement disorders, 2023-12, Vol.38 (12), p.2230-2240

2023

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RAN Translation in Huntington Disease

Use of single guided Cas9 nickase to facilitate precise and efficient genome editing in human iPSCs

Synaptic dysregulation in a human iPS cell model of mental disorders

Huntington disease: natural history, biomarkers and prospects for therapeutics

An Antisense CAG Repeat Transcript at JPH3 Locus Mediates Expanded Polyglutamine Protein Toxicity in Huntington's Disease-like 2 Mice

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

Prevalence of Psychotic Symptoms in a Community-Based Parkinson Disease Sample

Generation of a human induced pluripotent stem cell line JHUi004-A with heterozygous mutation for spinocerebellar ataxia type 12 using genome editing

Age, CAG repeat length, and clinical progression in Huntington's disease

A high-throughput screening to identify small molecules that suppress huntingtin promoter activity or activate huntingtin-antisense promoter activity

Generation of a human induced pluripotent stem cell line JHUi003-A with homozygous mutation for spinocerebellar ataxia type 12 using genome editing

Pharmacological rescue in patient iPSC and mouse models with a rare DISC1 mutation

Loss of junctophilin-3 contributes to huntington disease-like 2 pathogenesis

Author Correction: Pharmacological rescue in patient iPSC and mouse models with a rare DISC1 mutation

Phosphorodiamidate morpholino oligomers suppress mutant huntingtin expression and attenuate neurotoxicity

Analysis of differential gene expression mediated by clozapine in human postmortem brains

Emerging differences between Huntington's disease-like 2 and Huntington's disease: A comparison using MRI brain volumetry

A Systematic Review of the Huntington Disease-Like 2 Phenotype

Differential Changes in Functional Connectivity of Striatum-Prefrontal and Striatum-Motor Circuits in Premanifest Huntington’s Disease

Common SNP-Based Haplotype Analysis of the 4p16.3 Huntington Disease Gene Region

Pharmacologic Treatment of Anxiety Disorders in Parkinson Disease

Research Domain Criteria: Strengths, Weaknesses, and Potential Alternatives for Future Psychiatric Research

The timing and severity of clozapine-associated neutropenia in the US: Is the risk overstated?

Role of Bβ1 overexpression in the pathogenesis of SCA12

Bidirectional Transcription at the PPP2R2B Gene Locus in Spinocerebellar Ataxia Type 12

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