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Fetal gene therapy for neurodegenerative disease of infants

Nature medicine, 2018-09, Vol.24 (9), p.1317-1323

2018

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Disruption of the autophagy-lysosome pathway is involved in neuropathology of the nclf mouse model of neuronal ceroid lipofuscinosis

PloS one, 2012-04, Vol.7 (4), p.e35493

2012

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Intravenous administration of AAV2/9 to the fetal and neonatal mouse leads to differential targeting of CNS cell types and extensive transduction of the nervous system

The FASEB journal, 2011-10, Vol.25 (10), p.3505-3518

2011

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Early glial activation, synaptic changes and axonal pathology in the thalamocortical system of Niemann–Pick type C1 mice

Neurobiology of disease, 2012-03, Vol.45 (3), p.1086-1100

2012

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An anti-neuroinflammatory that targets dysregulated glia enhances the efficacy of CNS-directed gene therapy in murine infantile neuronal ceroid lipofuscinosis

The Journal of neuroscience, 2014-09, Vol.34 (39), p.13077-13082

2014

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Generation of light-producing somatic-transgenic mice using adeno-associated virus vectors

Scientific reports, 2020-02, Vol.10 (1), p.2121-2121, Article 2121

2020

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Glial cells are functionally impaired in juvenile neuronal ceroid lipofuscinosis and detrimental to neurons

Acta neuropathologica communications, 2017-10, Vol.5 (1), p.74-74, Article 74

2017

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Appalachian Nursing Homes: A Descriptive Analysis of Quality, Staffing, and Cost

Journal of the American Medical Directors Association, 2024-10, Vol.25 (10), p.105174, Article 105174

2024

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Systemic gene delivery following intravenous administration of AAV9 to fetal and neonatal mice and late‐gestation nonhuman primates

The FASEB journal, 2015-09, Vol.29 (9), p.3876-3888

2015

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DHHC5 Interacts with PDZ Domain 3 of Post-synaptic Density-95 (PSD-95) Protein and Plays a Role in Learning and Memory

The Journal of biological chemistry, 2010-04, Vol.285 (17), p.13022-13031

2010

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Intravenous high-dose enzyme replacement therapy with recombinant palmitoyl–protein thioesterase reduces visceral lysosomal storage and modestly prolongs survival in a preclinical mouse model of infantile neuronal ceroid lipofuscinosis

Molecular genetics and metabolism, 2012-09, Vol.107 (1-2), p.213-221

2012

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Regional Brain Atrophy in Mouse Models of Neuronal Ceroid Lipofuscinosis: A New Rostrocaudal Perspective

Journal of child neurology, 2013-09, Vol.28 (9), p.1117-1122

2013

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Non-invasive molecular imaging of inflammatory macrophages in allograft rejection

EJNMMI research, 2015-12, Vol.5 (1), p.69-69, Article 69

2015

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Bilateral Ischial Tuberosity Stress Fractures in an Adolescent Football Player: A Case Report

Clinical journal of sport medicine, 2023-03, Vol.33 (2), p.e16-e18

2023

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Exacerbated neuronal ceroid lipofuscinosis phenotype in Cln1/5 double-knockout mice

Disease models & mechanisms, 2013-03, Vol.6 (2), p.342-357

2013

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Behavioral deficits, early gliosis, dysmyelination and synaptic dysfunction in a mouse model of mucolipidosis IV

Acta neuropathologica communications, 2014-09, Vol.2 (1), p.133-133, Article 133

2014

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Proteomic mapping of differentially vulnerable pre-synaptic populations identifies regulators of neuronal stability in vivo

Scientific reports, 2017-09, Vol.7 (1), p.12412-18, Article 12412

2017

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Synergistic effects of central nervous system-directed gene therapy and bone marrow transplantation in the murine model of infantile neuronal ceroid lipofuscinosis

Annals of neurology, 2012-06, Vol.71 (6), p.797-804

2012

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Nonclinical evaluation of CNS-administered TPP1 enzyme replacement in canine CLN2 neuronal ceroid lipofuscinosis

Molecular genetics and metabolism, 2015-02, Vol.114 (2), p.281-293

2015

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Treatment of HCV in renal transplant patients with peginterferon and ribavirin: long-term follow-up

Clinical and experimental nephrology, 2017-10, Vol.21 (5), p.764-770

2017

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Screening Algorithm for BK Virus-Associated Nephropathy Using Sequential Testing of Urinary Cytology: A Probabilistic Model Analysis

American journal of nephrology, 2016-02, Vol.42 (6), p.410-417

2016

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Simultaneous Bilateral Quadriceps Tendon Rupture Secondary to Parathyroid Carcinoma: A Case Report

Journal of the American Academy of Orthopaedic Surgeons. Global research & reviews, 2023-11, Vol.7 (11)

2023

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Molecular correlates of axonal and synaptic pathology in mouse models of Batten disease

Human molecular genetics, 2009-11, Vol.18 (21), p.4066-4080

2009

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Combination small molecule PPT1 mimetic and CNS-directed gene therapy as a treatment for infantile neuronal ceroid lipofuscinosis

Journal of inherited metabolic disease, 2012-09, Vol.35 (5), p.847-857

2012

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Current therapies for the soluble lysosomal forms of neuronal ceroid lipofuscinosis

Biochemical Society transactions, 2010-12, Vol.38 (6), p.1484-1488

2010

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Fetal gene therapy for neurodegenerative disease of infants

Disruption of the autophagy-lysosome pathway is involved in neuropathology of the nclf mouse model of neuronal ceroid lipofuscinosis

Intravenous administration of AAV2/9 to the fetal and neonatal mouse leads to differential targeting of CNS cell types and extensive transduction of the nervous system

Early glial activation, synaptic changes and axonal pathology in the thalamocortical system of Niemann–Pick type C1 mice

An anti-neuroinflammatory that targets dysregulated glia enhances the efficacy of CNS-directed gene therapy in murine infantile neuronal ceroid lipofuscinosis

Generation of light-producing somatic-transgenic mice using adeno-associated virus vectors

Glial cells are functionally impaired in juvenile neuronal ceroid lipofuscinosis and detrimental to neurons

Appalachian Nursing Homes: A Descriptive Analysis of Quality, Staffing, and Cost

Systemic gene delivery following intravenous administration of AAV9 to fetal and neonatal mice and late‐gestation nonhuman primates

DHHC5 Interacts with PDZ Domain 3 of Post-synaptic Density-95 (PSD-95) Protein and Plays a Role in Learning and Memory

Intravenous high-dose enzyme replacement therapy with recombinant palmitoyl–protein thioesterase reduces visceral lysosomal storage and modestly prolongs survival in a preclinical mouse model of infantile neuronal ceroid lipofuscinosis

Regional Brain Atrophy in Mouse Models of Neuronal Ceroid Lipofuscinosis: A New Rostrocaudal Perspective

Non-invasive molecular imaging of inflammatory macrophages in allograft rejection

Bilateral Ischial Tuberosity Stress Fractures in an Adolescent Football Player: A Case Report

Exacerbated neuronal ceroid lipofuscinosis phenotype in Cln1/5 double-knockout mice

Behavioral deficits, early gliosis, dysmyelination and synaptic dysfunction in a mouse model of mucolipidosis IV

Proteomic mapping of differentially vulnerable pre-synaptic populations identifies regulators of neuronal stability in vivo

Synergistic effects of central nervous system-directed gene therapy and bone marrow transplantation in the murine model of infantile neuronal ceroid lipofuscinosis

Nonclinical evaluation of CNS-administered TPP1 enzyme replacement in canine CLN2 neuronal ceroid lipofuscinosis

Treatment of HCV in renal transplant patients with peginterferon and ribavirin: long-term follow-up

Screening Algorithm for BK Virus-Associated Nephropathy Using Sequential Testing of Urinary Cytology: A Probabilistic Model Analysis

Simultaneous Bilateral Quadriceps Tendon Rupture Secondary to Parathyroid Carcinoma: A Case Report

Molecular correlates of axonal and synaptic pathology in mouse models of Batten disease

Combination small molecule PPT1 mimetic and CNS-directed gene therapy as a treatment for infantile neuronal ceroid lipofuscinosis

Current therapies for the soluble lysosomal forms of neuronal ceroid lipofuscinosis

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