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Elimination of Toxic Microsatellite Repeat Expansion RNA by RNA-Targeting Cas9

Cell, 2017-08, Vol.170 (5), p.899-912.e10

2017

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Impeding Transcription of Expanded Microsatellite Repeats by Deactivated Cas9

Molecular cell, 2017-11, Vol.68 (3), p.479-490.e5

2017

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RAN Translation Regulated by Muscleblind Proteins in Myotonic Dystrophy Type 2

Neuron (Cambridge, Mass.), 2017-09, Vol.95 (6), p.1292-1305.e5

2017

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A CRISPR-Cas13a Based Strategy That Tracks and Degrades Toxic RNA in Myotonic Dystrophy Type 1

Frontiers in genetics, 2020-12, Vol.11, p.594576

2020

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Genome Therapy of Myotonic Dystrophy Type 1 iPS Cells for Development of Autologous Stem Cell Therapy

Molecular therapy, 2016-08, Vol.24 (8), p.1378-1387

2016

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Therapeutic Genome Editing for Myotonic Dystrophy Type 1 Using CRISPR/Cas9

Molecular therapy, 2018-11, Vol.26 (11), p.2617-2630

2018

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DNAzyme Cleavage of CAG Repeat RNA in Polyglutamine Diseases

Neurotherapeutics, 2021-07, Vol.18 (3), p.1710-1728

2021

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Genome Modification Leads to Phenotype Reversal in Human Myotonic Dystrophy Type 1 Induced Pluripotent Stem Cell‐Derived Neural Stem Cells

Stem cells (Dayton, Ohio), 2015-06, Vol.33 (6), p.1829-1838

2015

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Human iPSC Models to Study Orphan Diseases: Muscular Dystrophies

Current stem cell reports, 2018-12, Vol.4 (4), p.299-309

2018

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Dystonia and ataxia progression in spinocerebellar ataxias

Parkinsonism & related disorders, 2017-12, Vol.45, p.75-80

2017

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A knockin mouse model of spinocerebellar ataxia type 3 exhibits prominent aggregate pathology and aberrant splicing of the disease gene transcript

Human molecular genetics, 2015-03, Vol.24 (5), p.1211-1224

2015

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Generation of Human-Induced Pluripotent Stem Cells to Model Spinocerebellar Ataxia Type 2 In vitro

Journal of molecular neuroscience, 2013-10, Vol.51 (2), p.237-248

2013

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Purkinje cell loss is the major brain pathology of spinocerebellar ataxia type 10

Journal of neurology, neurosurgery and psychiatry, 2013-12, Vol.84 (12), p.1409-1411

2013

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The Initial Symptom and Motor Progression in Spinocerebellar Ataxias

Cerebellum (London, England), 2017-06, Vol.16 (3), p.615-622

2017

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Ataxia

Continuum (Minneapolis, Minn.), 2016-08, Vol.22 (4 Movement Disorders), p.1208

2016

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Receptor Tyrosine Kinase EphB4 Is a Survival Factor in Breast Cancer

The American journal of pathology, 2006-07, Vol.169 (1), p.279-293

2006

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Generation of neural cells from DM1 induced pluripotent stem cells as cellular model for the study of central nervous system neuropathogenesis

Cellular reprogramming, 2013-04, Vol.15 (2), p.166

2013

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Expression and Significance of Vascular Endothelial Growth Factor Receptor 2 in Bladder Cancer

The Journal of urology, 2006-04, Vol.175 (4), p.1245-1252

2006

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Malignant mesothelioma growth inhibition by agents that target the VEGF and VEGF‐C autocrine loops

International journal of cancer, 2003-05, Vol.104 (5), p.603-610

2003

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EphB4 expression and biological significance in prostate cancer

Cancer research (Chicago, Ill.), 2005-06, Vol.65 (11), p.4623-4632

2005

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Dynamic changes of nuclear RNA foci in proliferating DM1 cells

Histochemistry and cell biology, 2015-06, Vol.143 (6), p.557-564

2015

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Postural Tremor and Ataxia Progression in Spinocerebellar Ataxias

Tremor and other hyperkinetic movements (New York, N.Y.), 2017-10, Vol.7

2017

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EphB4 provides survival advantage to squamous cell carcinoma of the head and neck

International journal of cancer, 2006-09, Vol.119 (6), p.1236-1248

2006

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Ephrin B2 expression in Kaposi sarcoma is induced by human herpesvirus type 8: phenotype switch from venous to arterial endothelium

Blood, 2005-02, Vol.105 (3), p.1310-1318

2005

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Elevated Peripheral Myelin Protein 22, Reduced Mitotic Potential, and Proteasome Impairment in Dermal Fibroblasts from Charcot-Marie-Tooth Disease Type 1A Patients

The American journal of pathology, 2018-03, Vol.188 (3), p.728-738

2018

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Elimination of Toxic Microsatellite Repeat Expansion RNA by RNA-Targeting Cas9

Impeding Transcription of Expanded Microsatellite Repeats by Deactivated Cas9

RAN Translation Regulated by Muscleblind Proteins in Myotonic Dystrophy Type 2

A CRISPR-Cas13a Based Strategy That Tracks and Degrades Toxic RNA in Myotonic Dystrophy Type 1

Genome Therapy of Myotonic Dystrophy Type 1 iPS Cells for Development of Autologous Stem Cell Therapy

Therapeutic Genome Editing for Myotonic Dystrophy Type 1 Using CRISPR/Cas9

DNAzyme Cleavage of CAG Repeat RNA in Polyglutamine Diseases

Genome Modification Leads to Phenotype Reversal in Human Myotonic Dystrophy Type 1 Induced Pluripotent Stem Cell‐Derived Neural Stem Cells

Human iPSC Models to Study Orphan Diseases: Muscular Dystrophies

Dystonia and ataxia progression in spinocerebellar ataxias

A knockin mouse model of spinocerebellar ataxia type 3 exhibits prominent aggregate pathology and aberrant splicing of the disease gene transcript

Generation of Human-Induced Pluripotent Stem Cells to Model Spinocerebellar Ataxia Type 2 In vitro

Purkinje cell loss is the major brain pathology of spinocerebellar ataxia type 10

The Initial Symptom and Motor Progression in Spinocerebellar Ataxias

Ataxia

Receptor Tyrosine Kinase EphB4 Is a Survival Factor in Breast Cancer

Generation of neural cells from DM1 induced pluripotent stem cells as cellular model for the study of central nervous system neuropathogenesis

Expression and Significance of Vascular Endothelial Growth Factor Receptor 2 in Bladder Cancer

Malignant mesothelioma growth inhibition by agents that target the VEGF and VEGF‐C autocrine loops

EphB4 expression and biological significance in prostate cancer

Dynamic changes of nuclear RNA foci in proliferating DM1 cells

Postural Tremor and Ataxia Progression in Spinocerebellar Ataxias

EphB4 provides survival advantage to squamous cell carcinoma of the head and neck

Ephrin B2 expression in Kaposi sarcoma is induced by human herpesvirus type 8: phenotype switch from venous to arterial endothelium

Elevated Peripheral Myelin Protein 22, Reduced Mitotic Potential, and Proteasome Impairment in Dermal Fibroblasts from Charcot-Marie-Tooth Disease Type 1A Patients

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