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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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Overcoming the Blood-Brain Barrier with High-Dose Enzyme Replacement Therapy in Murine Mucopolysaccharidosis VII

Proceedings of the National Academy of Sciences - PNAS, 2005-10, Vol.102 (41), p.14777-14782

2005

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Chemically modified β-glucuronidase crosses blood-brain barrier and clears neuronal storage in murine mucopolysaccharidosis VII

Proceedings of the National Academy of Sciences - PNAS, 2008-02, Vol.105 (7), p.2616-2621

2008

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Biochemical evidence for superior correction of neuronal storage by chemically modified enzyme in murine mucopolysaccharidosis VII

Proceedings of the National Academy of Sciences - PNAS, 2012-10, Vol.109 (42), p.17022-17027

2012

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Glycosylation-Independent Targeting Enhances Enzyme Delivery to Lysosomes and Decreases Storage in Mucopolysaccharidosis Type VII Mice

Proceedings of the National Academy of Sciences - PNAS, 2004-03, Vol.101 (9), p.3083-3088

2004

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Neonatal Gene Transfer Leads to Widespread Correction of Pathology in a Murine Model of Lysosomal Storage Disease

Proceedings of the National Academy of Sciences - PNAS, 1999-03, Vol.96 (5), p.2296-2300

1999

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Brain-Directed Gene Therapy for Lysosomal Storage Disease: Going Well beyond the Blood-Brain Barrier

Proceedings of the National Academy of Sciences - PNAS, 2002-04, Vol.99 (9), p.5760-5762

2002

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Infused Fc-tagged β-glucuronidase crosses the placenta and produces clearance of storage in utero in mucopolysaccharidosis VII mice

Proceedings of the National Academy of Sciences - PNAS, 2008-06, Vol.105 (24), p.8375-8380

2008

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Enzyme Therapy in Mannose Receptor-Null Mucopolysaccharidosis VII Mice Defines Roles for the Mannose 6-Phosphate and Mannose Receptors

Proceedings of the National Academy of Sciences - PNAS, 2006-10, Vol.103 (41), p.15172-15177

2006

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Transgene Produces Massive Overexpression of Human β-Glucuronidase in Mice, Lysosomal Storage of Enzyme, and Strain-Dependent Tumors

Proceedings of the National Academy of Sciences - PNAS, 2003-03, Vol.100 (5), p.2669-2673

2003

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Adenovirus-Mediated Gene Transfer and Expression of Human β -glucuronidase Gene in the Liver, Spleen, and Central Nervous System in Mucopolysaccharidosis Type VII Mice

Proceedings of the National Academy of Sciences - PNAS, 1997-02, Vol.94 (4), p.1287-1292

1997

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Infused Fc-tagged [beta]-glucuronidase crosses the placenta and produces clearance of storage in utero in mucopolysaccharidosis VII mice

Proceedings of the National Academy of Sciences - PNAS, 2008-06, Vol.105 (24), p.8375

2008

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Active Site Mutant Transgene Confers Tolerance to Human β-Glucuronidase without Affecting the Phenotype of MPS VII Mice

Proceedings of the National Academy of Sciences - PNAS, 2001-02, Vol.98 (5), p.2205-2210

2001

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Chemically modified [beta]-glucuronidase crosses blood-brain barrier and clears neuronal storage in murine mucopolysaccharidosis VII

Proceedings of the National Academy of Sciences - PNAS, 2008-02, Vol.105 (7), p.2616

2008

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Brain-directed gene therapy for lyosomal storage disease: Going well beyond the blood-brain barrier

Proceedings of the National Academy of Sciences - PNAS, 2002-04, Vol.99 (9), p.5760

2002

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Missense Models [Gustm(E536A)Sly, Gustm(E536Q)Sly, and Gustm(L175F)Sly] of Murine Mucopolysaccharidosis Type VII Produced by Targeted Mutagenesis

Proceedings of the National Academy of Sciences - PNAS, 2002-11, Vol.99 (23), p.14982-14987

2002

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Correction of Murine Mucopolysaccharidosis VII by a Human β- Glucuronidase Transgene

Proceedings of the National Academy of Sciences - PNAS, 1990-05, Vol.87 (10), p.3914-3918

1990

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

Overcoming the Blood-Brain Barrier with High-Dose Enzyme Replacement Therapy in Murine Mucopolysaccharidosis VII

Chemically modified β-glucuronidase crosses blood-brain barrier and clears neuronal storage in murine mucopolysaccharidosis VII

Biochemical evidence for superior correction of neuronal storage by chemically modified enzyme in murine mucopolysaccharidosis VII

Glycosylation-Independent Targeting Enhances Enzyme Delivery to Lysosomes and Decreases Storage in Mucopolysaccharidosis Type VII Mice

Neonatal Gene Transfer Leads to Widespread Correction of Pathology in a Murine Model of Lysosomal Storage Disease

Brain-Directed Gene Therapy for Lysosomal Storage Disease: Going Well beyond the Blood-Brain Barrier

Infused Fc-tagged β-glucuronidase crosses the placenta and produces clearance of storage in utero in mucopolysaccharidosis VII mice

Enzyme Therapy in Mannose Receptor-Null Mucopolysaccharidosis VII Mice Defines Roles for the Mannose 6-Phosphate and Mannose Receptors

Transgene Produces Massive Overexpression of Human β-Glucuronidase in Mice, Lysosomal Storage of Enzyme, and Strain-Dependent Tumors

Adenovirus-Mediated Gene Transfer and Expression of Human β -glucuronidase Gene in the Liver, Spleen, and Central Nervous System in Mucopolysaccharidosis Type VII Mice

Infused Fc-tagged [beta]-glucuronidase crosses the placenta and produces clearance of storage in utero in mucopolysaccharidosis VII mice

Active Site Mutant Transgene Confers Tolerance to Human β-Glucuronidase without Affecting the Phenotype of MPS VII Mice

Chemically modified [beta]-glucuronidase crosses blood-brain barrier and clears neuronal storage in murine mucopolysaccharidosis VII

Brain-directed gene therapy for lyosomal storage disease: Going well beyond the blood-brain barrier

Missense Models [Gustm(E536A)Sly, Gustm(E536Q)Sly, and Gustm(L175F)Sly] of Murine Mucopolysaccharidosis Type VII Produced by Targeted Mutagenesis

Correction of Murine Mucopolysaccharidosis VII by a Human β- Glucuronidase Transgene

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