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A Mouse Model of Classical Late-Infantile Neuronal Ceroid Lipofuscinosis Based on Targeted Disruption of the CLN2 Gene Results in a Loss of Tripeptidyl-Peptidase I Activity and Progressive Neurodegeneration

The Journal of neuroscience, 2004-10, Vol.24 (41), p.9117-9126

2004

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Characterization of AAV-mediated dorsal root ganglionopathy

Molecular therapy. Methods & clinical development, 2022-03, Vol.24, p.342-354

2022

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Manganese Superoxide Dismutase Signals Matrix Metalloproteinase Expression via H2O2-dependent ERK1/2 Activation

The Journal of biological chemistry, 2001-04, Vol.276 (17), p.14264-14270

2001

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Epigenetic Modulation of Human Induced Pluripotent Stem Cell Differentiation to Oligodendrocytes

International journal of molecular sciences, 2016-04, Vol.17 (4), p.614-614

2016

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Transcriptomic Analysis of Induced Pluripotent Stem Cells Derived from Patients with Bipolar Disorder from an Old Order Amish Pedigree

PloS one, 2015-11, Vol.10 (11), p.e0142693-e0142693

2015

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Roles of p53 and p27 Kip1 in the regulation of neurogenesis in the murine adult subventricular zone

The European journal of neuroscience, 2011-10, Vol.34 (7), p.1040-1052

2011

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Comparative dose effectiveness of intravenous and intrathecal AAV9.CB7.hIDS, RGX-121, in mucopolysaccharidosis type II mice

Molecular therapy. Methods & clinical development, 2024-03, Vol.32 (1), p.101201-101201, Article 101201

2024

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Roles of p53 and p27(Kip1) in the regulation of neurogenesis in the murine adult subventricular zone

The European journal of neuroscience, 2011-10, Vol.34 (7), p.1040-1052

2011

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Residual levels of tripeptidyl-peptidase I activity dramatically ameliorate disease in late-infantile neuronal ceroid lipofuscinosis

Molecular genetics and metabolism, 2008-06, Vol.94 (2), p.222-233

2008

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Genetic modulation of apoptotic pathways fails to alter disease course in tripeptidyl-peptidase 1 deficient mice

Neuroscience letters, 2009-03, Vol.453 (1), p.27-30

2009

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Dipeptidyl-peptidase I does not functionally compensate for the loss of tripeptidyl-peptidase I in the neurodegenerative disease late-infantile neuronal ceroid lipofuscinosis

Biochemical journal, 2008-10, Vol.415 (2), p.225-232

2008

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pharmacology study using retina organoids and retina-on-a-chip of CLN2 patient-derived induced pluripotent stem cells

Molecular genetics and metabolism, 2023-02, Vol.138 (2), p.107184, Article 107184

2023

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In vitro pharmacology study using retina organoids and retina-on-a-chip of CLN2 patient-derived induced pluripotent stem cells

Molecular genetics and metabolism, 2023-02, Vol.138 (2)

2023

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Characterization of retinal degeneration phenotype in classic CLN2 disease using OCT biomarkers and an retinal model

Molecular genetics and metabolism, 2023-02, Vol.138 (2), p.107244, Article 107244

2023

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Characterization of retinal degeneration phenotype in classic CLN2 disease using OCT biomarkers and an in vitro retinal model

Molecular genetics and metabolism, 2023-02, Vol.138 (2)

2023

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RGX-381: Interim results from the first-in-human clinical trial of an investigational gene therapy for the treatment of ocular manifestations of CLN2 Batten disease

Molecular genetics and metabolism, 2024-02, Vol.141 (2), p.107987, Article 107987

2024

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Subretinal injection of RGX-381 to cynomolgus monkeys leads to supraphysiological levels of TPP1 in the eye

Molecular genetics and metabolism, 2021-02, Vol.132 (2), p.S21-S21

2021

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IDENTIFICATION OF MEASURABLE PHENOTYPES RELEVANT TO ALZHEIMER’S DISEASE USING HUMAN IPSC-DERIVED NEURONS

Alzheimer's & dementia, 2018-07, Vol.14 (7), p.P1130-P1130

2018

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P3‐163: IDENTIFICATION OF MEASURABLE PHENOTYPES RELEVANT TO ALZHEIMER'S DISEASE USING HUMAN IPSC‐DERIVED NEURONS

Alzheimer's & dementia, 2018-07, Vol.14 (7S_Part_21), p.P1130-P1130

2018

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Multiparametric assessment of networked electrical activity using induced pluripotent stem cell-derived glutamatergic neurons

Journal of pharmacological and toxicological methods, 2018-09, Vol.93, p.133-134

2018

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Roles of p53 and p27Kip1 in the regulation of neurogenesis in the murine adult subventricular zone

The European journal of neuroscience, 2011-10, Vol.34 (7), p.1040-1052

2011

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Roles of p53 and p27 Kip1 in the regulation of neurogenesis in the murine adult subventricular zone: Neurogenesis in the adult subventricular zone

The European journal of neuroscience, 2011-10, Vol.34 (7), p.1040-1052

2011

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Transcriptomic Analysis of Induced Pluripotent Stem Cells Derived from Patients with Bipolar Disorder from an Old Order Amish Pedigree: e0142693

PloS one, 2015-01, Vol.10 (11)

2015

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Potential mechanisms for the inhibition of tumor cell growth by manganese superoxide dismutase

Antioxidants & redox signaling, 2001-06, Vol.3 (3), p.361-373

2001

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Frequent inactivation of SPARC by promoter hypermethylation in colon cancers

International journal of cancer, 2007-08, Vol.121 (3), p.567-575

2007

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A Mouse Model of Classical Late-Infantile Neuronal Ceroid Lipofuscinosis Based on Targeted Disruption of the CLN2 Gene Results in a Loss of Tripeptidyl-Peptidase I Activity and Progressive Neurodegeneration

Characterization of AAV-mediated dorsal root ganglionopathy

Manganese Superoxide Dismutase Signals Matrix Metalloproteinase Expression via H2O2-dependent ERK1/2 Activation

Epigenetic Modulation of Human Induced Pluripotent Stem Cell Differentiation to Oligodendrocytes

Transcriptomic Analysis of Induced Pluripotent Stem Cells Derived from Patients with Bipolar Disorder from an Old Order Amish Pedigree

Roles of p53 and p27 Kip1 in the regulation of neurogenesis in the murine adult subventricular zone

Comparative dose effectiveness of intravenous and intrathecal AAV9.CB7.hIDS, RGX-121, in mucopolysaccharidosis type II mice

Roles of p53 and p27(Kip1) in the regulation of neurogenesis in the murine adult subventricular zone

Residual levels of tripeptidyl-peptidase I activity dramatically ameliorate disease in late-infantile neuronal ceroid lipofuscinosis

Genetic modulation of apoptotic pathways fails to alter disease course in tripeptidyl-peptidase 1 deficient mice

Dipeptidyl-peptidase I does not functionally compensate for the loss of tripeptidyl-peptidase I in the neurodegenerative disease late-infantile neuronal ceroid lipofuscinosis

pharmacology study using retina organoids and retina-on-a-chip of CLN2 patient-derived induced pluripotent stem cells

In vitro pharmacology study using retina organoids and retina-on-a-chip of CLN2 patient-derived induced pluripotent stem cells

Characterization of retinal degeneration phenotype in classic CLN2 disease using OCT biomarkers and an retinal model

Characterization of retinal degeneration phenotype in classic CLN2 disease using OCT biomarkers and an in vitro retinal model

RGX-381: Interim results from the first-in-human clinical trial of an investigational gene therapy for the treatment of ocular manifestations of CLN2 Batten disease

Subretinal injection of RGX-381 to cynomolgus monkeys leads to supraphysiological levels of TPP1 in the eye

IDENTIFICATION OF MEASURABLE PHENOTYPES RELEVANT TO ALZHEIMER’S DISEASE USING HUMAN IPSC-DERIVED NEURONS

P3‐163: IDENTIFICATION OF MEASURABLE PHENOTYPES RELEVANT TO ALZHEIMER'S DISEASE USING HUMAN IPSC‐DERIVED NEURONS

Multiparametric assessment of networked electrical activity using induced pluripotent stem cell-derived glutamatergic neurons

Roles of p53 and p27Kip1 in the regulation of neurogenesis in the murine adult subventricular zone

Roles of p53 and p27 Kip1 in the regulation of neurogenesis in the murine adult subventricular zone: Neurogenesis in the adult subventricular zone

Transcriptomic Analysis of Induced Pluripotent Stem Cells Derived from Patients with Bipolar Disorder from an Old Order Amish Pedigree: e0142693

Potential mechanisms for the inhibition of tumor cell growth by manganese superoxide dismutase

Frequent inactivation of SPARC by promoter hypermethylation in colon cancers

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