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Genome Editing in Human Pluripotent Stem Cells: Approaches, Pitfalls, and Solutions

Cell stem cell, 2016-01, Vol.18 (1), p.53-65

2016

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Dissecting the Causal Mechanism of X-Linked Dystonia-Parkinsonism by Integrating Genome and Transcriptome Assembly

Cell, 2018-02, Vol.172 (5), p.897-909.e21

2018

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A TALEN Genome-Editing System for Generating Human Stem Cell-Based Disease Models

Cell stem cell, 2013-02, Vol.12 (2), p.238-251

2013

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Schwann Cell to Axon Transfer of Ribosomes: Toward a Novel Understanding of the Role of Glia in the Nervous System

The Journal of neuroscience, 2008-10, Vol.28 (43), p.11024-11029

2008

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NFIL3 and cAMP Response Element-Binding Protein Form a Transcriptional Feedforward Loop that Controls Neuronal Regeneration-Associated Gene Expression

The Journal of neuroscience, 2009-12, Vol.29 (49), p.15542-15550

2009

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Decreased N-TAF1 expression in X-linked dystonia-parkinsonism patient-specific neural stem cells

Disease models & mechanisms, 2016-04, Vol.9 (4), p.451-462

2016

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Phosphatidic acid mediates demyelination in Lpin1 mutant mice

Genes & development, 2008-06, Vol.22 (12), p.1647-1661

2008

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Differential effects of lentiviral vector-mediated overexpression of nerve growth factor and glial cell line-derived neurotrophic factor on regenerating sensory and motor axons in the transected peripheral nerve

The European journal of neuroscience, 2008-10, Vol.28 (8), p.1467-1479

2008

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Modification of superoxide dismutase 1 (SOD1) properties by a GFP tag--implications for research into amyotrophic lateral sclerosis (ALS)

PloS one, 2010-03, Vol.5 (3), p.e9541

2010

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TALEN‐ and CRISPR/Cas9‐Mediated Gene Editing in Human Pluripotent Stem Cells Using Lipid‐Based Transfection

Current Protocols in Stem Cell Biology, 2015-08, Vol.34 (1), p.5B.3.1-5B.3.25

2015

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Disease onset in X-linked dystonia-parkinsonism correlates with expansion of a hexameric repeat within an SVA retrotransposon in TAF1

Proceedings of the National Academy of Sciences - PNAS, 2017-12, Vol.114 (51), p.E11020-E11028

2017

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The importance of transgene and cell type on the regeneration of adult retinal ganglion cell axons within reconstituted bridging grafts

Experimental neurology, 2007-10, Vol.207 (2), p.314-328

2007

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Morphological evidence for a transport of ribosomes from Schwann cells to regenerating axons

Glia, 2011-10, Vol.59 (10), p.1529-1539

2011

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Dystonia-specific mutations in THAP1 alter transcription of genes associated with neurodevelopment and myelin

American journal of human genetics, 2021-11, Vol.108 (11), p.2145-2158

2021

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Stem Cells Engineered During Different Stages of Reprogramming Reveal Varying Therapeutic Efficacies

Stem cells (Dayton, Ohio), 2018-06, Vol.36 (6), p.932-942

2018

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Lentiviral vector-mediated reporter gene expression in avulsed spinal ventral root is short-term, but is prolonged using an immune "stealth" transgene

Restorative neurology and neuroscience, 2007, Vol.25 (5-6), p.585

2007

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Neuroregenerative effects of lentiviral vector-mediated GDNF expression in reimplanted ventral roots

Molecular and cellular neuroscience, 2008-09, Vol.39 (1), p.105-117

2008

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Variation in TAF1 expression in female carrier induced pluripotent stem cells and human brain ontogeny has implications for adult neostriatum vulnerability in X-linked Dystonia Parkinsonism

eNeuro, 2022-07, Vol.9 (4), p.ENEURO.0129-22.2022

2022

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Profound differences in spontaneous long-term functional recovery after defined spinal tract lesions in the rat

Journal of neurotrauma, 2006-01, Vol.23 (1), p.18-35

2006

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X-linked Dystonia-Parkinsonism patient cells exhibit altered signaling via nuclear factor-kappa B

Neurobiology of disease, 2017-04, Vol.100, p.108-118

2017

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Therapeutic Neural Stem Cells Engineered during different stages of Reprogramming reveal varying Therapeutic efficacies

Stem cells (Dayton, Ohio), 2018-04, Vol.36 (6), p.932-942

2018

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Lentiviral-mediated transfer of CNTF to schwann cells within reconstructed peripheral nerve grafts enhances adult retinal ganglion cell survival and axonal regeneration

Molecular therapy, 2005-06, Vol.11 (6), p.906-915

2005

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Viral vector-mediated gene transfer of neurotrophins to promote regeneration of the injured spinal cord

Progress in Brain Research, 2004, Vol.146, p.451-476

2004

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A TALEN genome editing system to generate human stem cell-based disease models

Cell stem cell, 2012-12, Vol.12 (2), p.238-251

2012

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Genome Editing in Human Pluripotent Stem Cells: Approaches, Pitfalls, and Solutions

Dissecting the Causal Mechanism of X-Linked Dystonia-Parkinsonism by Integrating Genome and Transcriptome Assembly

A TALEN Genome-Editing System for Generating Human Stem Cell-Based Disease Models

Schwann Cell to Axon Transfer of Ribosomes: Toward a Novel Understanding of the Role of Glia in the Nervous System

SVA insertion in X-linked Dystonia Parkinsonism alters histone H3 acetylation associated with TAF1 gene

NFIL3 and cAMP Response Element-Binding Protein Form a Transcriptional Feedforward Loop that Controls Neuronal Regeneration-Associated Gene Expression

Decreased N-TAF1 expression in X-linked dystonia-parkinsonism patient-specific neural stem cells

Phosphatidic acid mediates demyelination in Lpin1 mutant mice

Differential effects of lentiviral vector-mediated overexpression of nerve growth factor and glial cell line-derived neurotrophic factor on regenerating sensory and motor axons in the transected peripheral nerve

Modification of superoxide dismutase 1 (SOD1) properties by a GFP tag--implications for research into amyotrophic lateral sclerosis (ALS)

TALEN‐ and CRISPR/Cas9‐Mediated Gene Editing in Human Pluripotent Stem Cells Using Lipid‐Based Transfection

Disease onset in X-linked dystonia-parkinsonism correlates with expansion of a hexameric repeat within an SVA retrotransposon in TAF1

The importance of transgene and cell type on the regeneration of adult retinal ganglion cell axons within reconstituted bridging grafts

Morphological evidence for a transport of ribosomes from Schwann cells to regenerating axons

Dystonia-specific mutations in THAP1 alter transcription of genes associated with neurodevelopment and myelin

Stem Cells Engineered During Different Stages of Reprogramming Reveal Varying Therapeutic Efficacies

Lentiviral vector-mediated reporter gene expression in avulsed spinal ventral root is short-term, but is prolonged using an immune "stealth" transgene

Neuroregenerative effects of lentiviral vector-mediated GDNF expression in reimplanted ventral roots

Variation in TAF1 expression in female carrier induced pluripotent stem cells and human brain ontogeny has implications for adult neostriatum vulnerability in X-linked Dystonia Parkinsonism

Profound differences in spontaneous long-term functional recovery after defined spinal tract lesions in the rat

X-linked Dystonia-Parkinsonism patient cells exhibit altered signaling via nuclear factor-kappa B

Therapeutic Neural Stem Cells Engineered during different stages of Reprogramming reveal varying Therapeutic efficacies

Lentiviral-mediated transfer of CNTF to schwann cells within reconstructed peripheral nerve grafts enhances adult retinal ganglion cell survival and axonal regeneration

Viral vector-mediated gene transfer of neurotrophins to promote regeneration of the injured spinal cord

A TALEN genome editing system to generate human stem cell-based disease models

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