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AAV Vector Integration Sites in Mouse Hepatocellular Carcinoma

Science (American Association for the Advancement of Science), 2007-07, Vol.317 (5837), p.477-477

2007

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A novel antibody to treat SOD1-related amyotrophic lateral sclerosis

Molecular therapy. Methods & clinical development, 2023-06, Vol.29, p.236-237

2023

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Lentiviral-Induced Spinal Cord Gliomas in Rat Model

International journal of molecular sciences, 2021-11, Vol.22 (23), p.12943

2021

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ATP7A Gene Addition to the Choroid Plexus Results in Long-term Rescue of the Lethal Copper Transport Defect in a Menkes Disease Mouse Model

Molecular therapy, 2011-12, Vol.19 (12), p.2114-2123

2011

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Altered intracellular localization and valosin-containing protein (p97 VCP) interaction underlie ATP7A-related distal motor neuropathy

Human molecular genetics, 2012-04, Vol.21 (8), p.1794-1807

2012

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Molecular correlates of epilepsy in early diagnosed and treated Menkes disease

Journal of inherited metabolic disease, 2010-10, Vol.33 (5), p.583-589

2010

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Translational read-through of a nonsense mutation in ATP7A impacts treatment outcome in Menkes disease

Annals of neurology, 2009-01, Vol.65 (1), p.108-113

2009

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Gene, Stem Cell, and Alternative Therapies for SCA 1

Frontiers in molecular neuroscience, 2016-08, Vol.9, p.67-67

2016

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Fetal Brain-directed AAV Gene Therapy Results in Rapid, Robust, and Persistent Transduction of Mouse Choroid Plexus Epithelia

Molecular therapy. Nucleic acids, 2013, Vol.2 (6), p.e101-e101, Article e101

2013

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Molecular and biochemical characterization of a unique mutation in CCS, the human copper chaperone to superoxide dismutase

Human mutation, 2012-08, Vol.33 (8), p.1207-1215

2012

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Molecular and Cellular Therapies for Motor Neuron Diseases

1, 2017

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Current and emerging targeted therapies for spinal muscular atrophy

Expert review of neurotherapeutics, 2023-12, Vol.23 (12), p.1189-1199

2023

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Progress in gene and cell therapies for the neuronal ceroid lipofuscinoses

Expert opinion on biological therapy, 2018-07, Vol.18 (7), p.755-764

2018

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Intrathecal Vector Delivery in Juvenile Rats via Lumbar Cistern Injection

Journal of visualized experiments, 2024-03 (205)

2024

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Minimally Invasive Injection to the Phrenic Nerve in a Porcine Hemidiaphragmatic Paralysis Model: A Pilot Study

Neurosurgery, 2020-10, Vol.87 (4), p.847-853

2020

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Neonatal Diagnosis and Treatment of Menkes Disease

The New England journal of medicine, 2008-02, Vol.358 (6), p.605-614

2008

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Gene Therapy for the Treatment of Neurological Disorders: Amyotrophic Lateral Sclerosis

Methods in molecular biology (Clifton, N.J.), 2016, Vol.1382, p.399

2016

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Controlling the Release of Neurotrophin‐3 and Chondroitinase ABC Enhances the Efficacy of Nerve Guidance Conduits

Advanced healthcare materials, 2020-07, Vol.9 (14), p.e2000200-n/a

2020

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Lot-to-Lot Variation in Adeno-Associated Virus Serotype 9 (AAV9) Preparations

Human gene therapy. Part B. Methods, 2019-12, Vol.30 (6), p.214-225

2019

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Central Nervous System-directed AAV2/5-Mediated Gene Therapy Synergizes with Bone Marrow Transplantation in the Murine Model of Globoid-cell Leukodystrophy

Molecular therapy, 2007-01, Vol.15 (1), p.44-52

2007

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Somatic mosaicism in Menkes disease suggests choroid plexus-mediated copper transport to the developing brain

American journal of medical genetics. Part A, 2010-10, Vol.152A (10), p.2529-2534

2010

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L-Threo-Dihydroxyphenylserine corrects neurochemical abnormalities in a menkes disease mouse model

Annals of neurology, 2013-02, Vol.73 (2), p.259-265

2013

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L-DOPS corrects neurochemical abnormalities in a Menkes disease mouse model

Annals of neurology, 2012-12, Vol.73 (2), p.259-265

2012

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Clinical outcomes in Menkes disease patients with a copper-responsive ATP7A mutation, G727R

Molecular genetics and metabolism, 2008-11, Vol.95 (3), p.174-181

2008

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Erratum to: Somatic Mosaicism in Menkes Disease Suggests Choroid Plexus-Mediated Copper Transport to the Developing Brain

American journal of medical genetics. Part A, 2011-08, Vol.155A (8), p.2044-2044

2011

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AAV Vector Integration Sites in Mouse Hepatocellular Carcinoma

A novel antibody to treat SOD1-related amyotrophic lateral sclerosis

Lentiviral-Induced Spinal Cord Gliomas in Rat Model

ATP7A Gene Addition to the Choroid Plexus Results in Long-term Rescue of the Lethal Copper Transport Defect in a Menkes Disease Mouse Model

Altered intracellular localization and valosin-containing protein (p97 VCP) interaction underlie ATP7A-related distal motor neuropathy

Molecular correlates of epilepsy in early diagnosed and treated Menkes disease

Translational read-through of a nonsense mutation in ATP7A impacts treatment outcome in Menkes disease

Gene, Stem Cell, and Alternative Therapies for SCA 1

Fetal Brain-directed AAV Gene Therapy Results in Rapid, Robust, and Persistent Transduction of Mouse Choroid Plexus Epithelia

Molecular and biochemical characterization of a unique mutation in CCS, the human copper chaperone to superoxide dismutase

Molecular and Cellular Therapies for Motor Neuron Diseases

Current and emerging targeted therapies for spinal muscular atrophy

Progress in gene and cell therapies for the neuronal ceroid lipofuscinoses

Intrathecal Vector Delivery in Juvenile Rats via Lumbar Cistern Injection

Minimally Invasive Injection to the Phrenic Nerve in a Porcine Hemidiaphragmatic Paralysis Model: A Pilot Study

Neonatal Diagnosis and Treatment of Menkes Disease

Gene Therapy for the Treatment of Neurological Disorders: Amyotrophic Lateral Sclerosis

Controlling the Release of Neurotrophin‐3 and Chondroitinase ABC Enhances the Efficacy of Nerve Guidance Conduits

Lot-to-Lot Variation in Adeno-Associated Virus Serotype 9 (AAV9) Preparations

Central Nervous System-directed AAV2/5-Mediated Gene Therapy Synergizes with Bone Marrow Transplantation in the Murine Model of Globoid-cell Leukodystrophy

Somatic mosaicism in Menkes disease suggests choroid plexus-mediated copper transport to the developing brain

L-Threo-Dihydroxyphenylserine corrects neurochemical abnormalities in a menkes disease mouse model

L-DOPS corrects neurochemical abnormalities in a Menkes disease mouse model

Clinical outcomes in Menkes disease patients with a copper-responsive ATP7A mutation, G727R

Erratum to: Somatic Mosaicism in Menkes Disease Suggests Choroid Plexus-Mediated Copper Transport to the Developing Brain

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