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Genetic ablation of acid ceramidase in Krabbe disease confirms the psychosine hypothesis and identifies a new therapeutic target

Proceedings of the National Academy of Sciences - PNAS, 2019-10, Vol.116 (40), p.20097-20103

2019

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Lentiviral‐Transduced Human Mesenchymal Stem Cells Persistently Express Therapeutic Levels of Enzyme in a Xenotransplantation Model of Human Disease

Stem cells (Dayton, Ohio), 2008-07, Vol.26 (7), p.1713-1722

2008

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Oxidative stress as a therapeutic target in globoid cell leukodystrophy

Experimental neurology, 2012-10, Vol.237 (2), p.444-452

2012

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Deceptively benign low-grade fibromyxoid sarcoma: array-comparative genomic hybridization decodes the diagnosis

Human pathology, 2013, Vol.44 (1), p.145-150

2013

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Enhanced Efficacy and Increased Long-Term Toxicity of CNS-Directed, AAV-Based Combination Therapy for Krabbe Disease

Molecular therapy, 2021-02, Vol.29 (2), p.691-701

2021

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Bone marrow transplantation augments the effect of brain- and spinal cord-directed adeno-associated virus 2/5 gene therapy by altering inflammation in the murine model of globoid-cell leukodystrophy

The Journal of neuroscience, 2011-07, Vol.31 (27), p.9945-9957

2011

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Replacing the enzyme alpha-L-iduronidase at birth ameliorates symptoms in the brain and periphery of dogs with mucopolysaccharidosis type I

Science translational medicine, 2010-12, Vol.2 (60), p.60ra89

2010

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Reversal of pathology in murine mucopolysaccharidosis type VII by somatic cell gene transfer

Nature (London), 1992-12, Vol.360 (6406), p.749-753

1992

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Early versus late treatment of spinal cord compression with long-term intrathecal enzyme replacement therapy in canine mucopolysaccharidosis type I

Molecular genetics and metabolism, 2010-10, Vol.101 (2-3), p.115-122

2010

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Attenuation of murine lysosomal storage disease by allogeneic neonatal bone marrow transplantation using costimulatory blockade and donor lymphocyte infusion without myeloablation

Clinical Immunology, 2006-05, Vol.119 (2), p.166-179

2006

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Intravitreal Gene Therapy Reduces Lysosomal Storage in Specific Areas of the CNS in Mucopolysaccharidosis VII Mice

The Journal of neuroscience, 2003-04, Vol.23 (8), p.3302-3307

2003

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Intracranial Injection of Recombinant Adeno-associated Virus Improves Cognitive Function in a Murine Model of Mucopolysaccharidosis Type VII

Molecular therapy, 2001-03, Vol.3 (3), p.351-358

2001

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Donor cell expansion is delayed following nonablative in utero transplantation to treat murine mucopolysaccharidosis type VII

Experimental hematology, 2003-11, Vol.31 (11), p.1112-1118

2003

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Nonablative neonatal marrow transplantation attenuates functional and physical defects of β-glucuronidase deficiency

Blood, 2001-03, Vol.97 (5), p.1498-1504

2001

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Retinal Function is Improved in a Murine Model of a Lysosomal Storage Disease Following Bone Marrow Transplantation

Experimental eye research, 2000-11, Vol.71 (5), p.469-481

2000

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Enzyme replacement therapy improves reproductive performance in mucopolysaccharidosis type VII mice but does not prevent postnatal losses

Pediatric research, 1999-02, Vol.45 (2), p.180-186

1999

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Biodistribution, kinetics, and efficacy of highly phosphorylated and non-phosphorylated beta-glucuronidase in the murine model of mucopolysaccharidosis VII

The Journal of biological chemistry, 2001-11, Vol.276 (46), p.43160-43165

2001

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Valproate Use Associated with Persistent Hyperammonemia and Mitochondrial Injury in a Child with Downʼs Syndrome

Journal of pediatric gastroenterology and nutrition, 1992-02, Vol.14 (2), p.223-227

1992

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Behavior and Therapeutic Efficacy of β-Glucuronidase–Positive Mononuclear Phagocytes in a Murine Model of Mucopolysaccharidosis Type VII

Blood, 1999-09, Vol.94 (6), p.2142-2150

1999

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Increased Life Span and Correction of Metabolic Defects in Murine Mucopolysaccharidosis Type VII After Syngeneic Bone Marrow Transplantation

Blood, 1991-12, Vol.78 (11), p.3081-3092

1991

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A genetically myeloablated MPS VII model detects the expansion and curative properties of as few as 100 enriched murine stem cells

Experimental hematology, 1999-11, Vol.27 (11), p.1691-1704

1999

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Biodistribution, Kinetics, and Efficacy of Highly Phosphorylated and Non-phosphorylated Î²-Glucuronidase in the Murine Model of Mucopolysaccharidosis VII

The Journal of biological chemistry, 2001-11, Vol.276 (46), p.43160

2001

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Toxoplasmic polymyositis revisited: case report and review of literature

Neuromuscular disorders : NMD, 1997-09, Vol.7 (6-7), p.390-396

1997

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Transplanted ER-MP12 hi20 −58 med/hi myeloid progenitors produce resident macrophages from marrow that are therapeutic for lysosomal storage disease

Blood cells, molecules, & diseases, 2004, Vol.32 (1), p.199-213

2004

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Transplanted ER-MP12hi20-58med/hi myeloid progenitors produce resident macrophages from marrow that are therapeutic for lysosomal storage disease

Blood cells, molecules, & diseases, 2004-01, Vol.32 (1), p.199-213

2004

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Genetic ablation of acid ceramidase in Krabbe disease confirms the psychosine hypothesis and identifies a new therapeutic target

Lentiviral‐Transduced Human Mesenchymal Stem Cells Persistently Express Therapeutic Levels of Enzyme in a Xenotransplantation Model of Human Disease

Oxidative stress as a therapeutic target in globoid cell leukodystrophy

Deceptively benign low-grade fibromyxoid sarcoma: array-comparative genomic hybridization decodes the diagnosis

Enhanced Efficacy and Increased Long-Term Toxicity of CNS-Directed, AAV-Based Combination Therapy for Krabbe Disease

Bone marrow transplantation augments the effect of brain- and spinal cord-directed adeno-associated virus 2/5 gene therapy by altering inflammation in the murine model of globoid-cell leukodystrophy

Replacing the enzyme alpha-L-iduronidase at birth ameliorates symptoms in the brain and periphery of dogs with mucopolysaccharidosis type I

Reversal of pathology in murine mucopolysaccharidosis type VII by somatic cell gene transfer

Early versus late treatment of spinal cord compression with long-term intrathecal enzyme replacement therapy in canine mucopolysaccharidosis type I

Attenuation of murine lysosomal storage disease by allogeneic neonatal bone marrow transplantation using costimulatory blockade and donor lymphocyte infusion without myeloablation

Intravitreal Gene Therapy Reduces Lysosomal Storage in Specific Areas of the CNS in Mucopolysaccharidosis VII Mice

Intracranial Injection of Recombinant Adeno-associated Virus Improves Cognitive Function in a Murine Model of Mucopolysaccharidosis Type VII

Donor cell expansion is delayed following nonablative in utero transplantation to treat murine mucopolysaccharidosis type VII

Nonablative neonatal marrow transplantation attenuates functional and physical defects of β-glucuronidase deficiency

Retinal Function is Improved in a Murine Model of a Lysosomal Storage Disease Following Bone Marrow Transplantation

Enzyme replacement therapy improves reproductive performance in mucopolysaccharidosis type VII mice but does not prevent postnatal losses

Biodistribution, kinetics, and efficacy of highly phosphorylated and non-phosphorylated beta-glucuronidase in the murine model of mucopolysaccharidosis VII

Valproate Use Associated with Persistent Hyperammonemia and Mitochondrial Injury in a Child with Downʼs Syndrome

Behavior and Therapeutic Efficacy of β-Glucuronidase–Positive Mononuclear Phagocytes in a Murine Model of Mucopolysaccharidosis Type VII

Increased Life Span and Correction of Metabolic Defects in Murine Mucopolysaccharidosis Type VII After Syngeneic Bone Marrow Transplantation

A genetically myeloablated MPS VII model detects the expansion and curative properties of as few as 100 enriched murine stem cells

Biodistribution, Kinetics, and Efficacy of Highly Phosphorylated and Non-phosphorylated Î²-Glucuronidase in the Murine Model of Mucopolysaccharidosis VII

Toxoplasmic polymyositis revisited: case report and review of literature

Transplanted ER-MP12 hi20 −58 med/hi myeloid progenitors produce resident macrophages from marrow that are therapeutic for lysosomal storage disease

Transplanted ER-MP12hi20-58med/hi myeloid progenitors produce resident macrophages from marrow that are therapeutic for lysosomal storage disease

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