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Dysfunction of endocytic and autophagic pathways in a lysosomal storage disease

Annals of neurology, 2006-04, Vol.59 (4), p.700-708

2006

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Enzyme replacement therapy in the mouse model of Pompe disease

Molecular genetics and metabolism, 2003-09, Vol.80 (1), p.159-169

2003

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Dysferlin Deficiency Enhances Monocyte Phagocytosis: A Model for the Inflammatory Onset of Limb-Girdle Muscular Dystrophy 2B

The American journal of pathology, 2008-03, Vol.172 (3), p.774-785

2008

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Replacing acid α-glucosidase in Pompe disease: recombinant and transgenic enzymes are equipotent, but neither completely clears glycogen from type II muscle fibers

Molecular therapy, 2005-01, Vol.11 (1), p.48-56

2005

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Correction of the Enzymatic and Functional Deficits in a Model of Pompe Disease Using Adeno-associated Virus Vectors

Molecular therapy, 2002-05, Vol.5 (5), p.571-578

2002

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Conditional up-Regulation of MHC Class I in Skeletal Muscle Leads to Self-Sustaining Autoimmune Myositis and Myositis-Specific Autoantibodies

Proceedings of the National Academy of Sciences - PNAS, 2000-08, Vol.97 (16), p.9209-9214

2000

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A variety of cytokines and immunologically relevant surface molecules are expressed by normal human skeletal muscle cells under proinflammatory stimuli

Clinical and experimental immunology, 1998-09, Vol.113 (3), p.407-414

1998

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Conditional tissue-specific expression of the acid α-glucosidase (GAA) gene in the GAA knockout mice : implications for therapy

Human molecular genetics, 2001-09, Vol.10 (19), p.2039-2047

2001

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Glycogen Stored in Skeletal but Not in Cardiac Muscle in Acid α-Glucosidase Mutant (Pompe) Mice Is Highly Resistant to Transgene-Encoded Human Enzyme

Molecular therapy, 2002-11, Vol.6 (5), p.601-608

2002

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Targeted disruption of the acid alpha-glucosidase gene in mice causes an illness with critical features of both infantile and adult human glycogen storage disease type II

The Journal of biological chemistry, 1998-07, Vol.273 (30), p.19086-19092

1998

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Animal models of myositis

Rheumatic diseases clinics of North America, 2002-11, Vol.28 (4), p.917-933

2002

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Modulation of disease severity in mice with targeted disruption of the acid α-glucosidase gene

Neuromuscular disorders : NMD, 2000-06, Vol.10 (4), p.283-291

2000

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The Role of an Autoantigen, Histidyl-tRNA Synthetase, in the Induction and Maintenance of Autoimmunity

Proceedings of the National Academy of Sciences - PNAS, 1990-12, Vol.87 (24), p.9933-9937

1990

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Induction of tolerance to a recombinant human enzyme, acid alpha-glucosidase, in enzyme deficient knockout mice

Transgenic research, 2003-04, Vol.12 (2), p.171-178

2003

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Activation of the endoplasmic reticulum stress response in autoimmune myositis: Potential role in muscle fiber damage and dysfunction

Arthritis and rheumatism, 2005-06, Vol.52 (6), p.1824-1835

2005

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Dysfunction of endocytic and autophagic pathways in a lysosomal storage disease

Enzyme replacement therapy in the mouse model of Pompe disease

Dysferlin Deficiency Enhances Monocyte Phagocytosis: A Model for the Inflammatory Onset of Limb-Girdle Muscular Dystrophy 2B

Replacing acid α-glucosidase in Pompe disease: recombinant and transgenic enzymes are equipotent, but neither completely clears glycogen from type II muscle fibers

Correction of the Enzymatic and Functional Deficits in a Model of Pompe Disease Using Adeno-associated Virus Vectors

Conditional up-Regulation of MHC Class I in Skeletal Muscle Leads to Self-Sustaining Autoimmune Myositis and Myositis-Specific Autoantibodies

A variety of cytokines and immunologically relevant surface molecules are expressed by normal human skeletal muscle cells under proinflammatory stimuli

Conditional tissue-specific expression of the acid α-glucosidase (GAA) gene in the GAA knockout mice : implications for therapy

Glycogen Stored in Skeletal but Not in Cardiac Muscle in Acid α-Glucosidase Mutant (Pompe) Mice Is Highly Resistant to Transgene-Encoded Human Enzyme

Targeted disruption of the acid alpha-glucosidase gene in mice causes an illness with critical features of both infantile and adult human glycogen storage disease type II

Animal models of myositis

Modulation of disease severity in mice with targeted disruption of the acid α-glucosidase gene

The Role of an Autoantigen, Histidyl-tRNA Synthetase, in the Induction and Maintenance of Autoimmunity

Induction of tolerance to a recombinant human enzyme, acid alpha-glucosidase, in enzyme deficient knockout mice

Activation of the endoplasmic reticulum stress response in autoimmune myositis: Potential role in muscle fiber damage and dysfunction

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