(Q45595051)

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Widespread correction of lysosomal storage following intrahepatic injection of a recombinant adeno-associated virus in the adult MPS VII mouse

scientific article published in September 2004

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Europe PubMed Central

14 December 2017

Widespread correction of lysosomal storage following intrahepatic injection of a recombinant adeno-associated virus in the adult MPS VII mouse (English)

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Europe PubMed Central

14 December 2017

author name string

Thomas J Sferra

1

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Europe PubMed Central

14 December 2017

Kristin Backstrom

2

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Europe PubMed Central

14 December 2017

Chuansong Wang

3

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Europe PubMed Central

14 December 2017

Rachel Rennard

4

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Europe PubMed Central

14 December 2017

Matt Miller

5

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Europe PubMed Central

14 December 2017

Yan Hu

6

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Europe PubMed Central

14 December 2017

publication date

1 September 2004

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Europe PubMed Central

14 December 2017

Molecular Therapy

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Europe PubMed Central

14 December 2017

10

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14 December 2017

3

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Europe PubMed Central

14 December 2017

478-491

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Europe PubMed Central

14 December 2017

Hematopoietic cell transplantation for inherited metabolic diseases: an overview of outcomes and practice guidelines

1 reference

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Beta glucuronidase deficiency: report of clinical, radiologic, and biochemical features of a new mucopolysaccharidosis

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Murine mucopolysaccharidosis type VII. Characterization of a mouse with beta-glucuronidase deficiency

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Murine mucopolysaccharidosis VIL: impact of therapies on the phenotype, clinical course, and pathology in a model of a lysosomal storage disease

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A single-base-pair deletion in the beta-glucuronidase gene accounts for the phenotype of murine mucopolysaccharidosis type VII

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Prevention of systemic clinical disease in MPS VII mice following AAV-mediated neonatal gene transfer

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Structure and function of the mannose 6-phosphate/insulinlike growth factor II receptors

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Mannose 6-phosphate receptors in sorting and transport of lysosomal enzymes

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Delivery of a retroviral vector expressing human beta-glucuronidase to the liver and spleen decreases lysosomal storage in mucopolysaccharidosis VII mice

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In vivo treatment of hemophilia A and mucopolysaccharidosis type VII using nonprimate lentiviral vectors.

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Evaluation of pathological manifestations of disease in mucopolysaccharidosis VII mice after neonatal hepatic gene therapy.

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Adenovirus-mediated gene transfer and expression of human beta-glucuronidase gene in the liver, spleen, and central nervous system in mucopolysaccharidosis type VII mice

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Adenovirus-mediated gene therapy for mucopolysaccharidosis VII: involvement of cross-correction in wide-spread distribution of the gene products and long-term effects of CTLA-4Ig coexpression

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The HIV Tat protein transduction domain improves the biodistribution of beta-glucuronidase expressed from recombinant viral vectors.

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Prolongation of transgene expression by coexpression of cytokine response modifier a in rodent liver after adenoviral gene transfer

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Treatment of lysosomal storage disease in MPS VII mice using a recombinant adeno-associated virus

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7 January 2021

Neonatal gene transfer leads to widespread correction of pathology in a murine model of lysosomal storage disease.

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Neonatal intramuscular injection with recombinant adeno-associated virus results in prolonged beta-glucuronidase expression in situ and correction of liver pathology in mucopolysaccharidosis type VII mice.

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Highly purified recombinant adeno-associated virus vectors are biologically active and free of detectable helper and wild-type viruses

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7 January 2021

Recombinant adeno-associated virus-mediated correction of lysosomal storage within the central nervous system of the adult mucopolysaccharidosis type VII mouse

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Treatment of a lysosomal storage disease, mucopolysaccharidosis VII, with microencapsulated recombinant cells

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Factors influencing in vivo transduction by recombinant adeno-associated viral vectors expressing the human factor IX cDNA.

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Improved hepatic gene transfer by using an adeno-associated virus serotype 5 vector

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Preclinical in vivo evaluation of pseudotyped adeno-associated virus vectors for liver gene therapy

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Decreased lysosomal storage in the adult MPS VII mouse brain in the vicinity of grafts of retroviral vector-corrected fibroblasts secreting high levels of beta-glucuronidase

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Retroviral gene transfer into the intestinal epithelium

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Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors

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Nonrandom transduction of recombinant adeno-associated virus vectors in mouse hepatocytes in vivo: cell cycling does not influence hepatocyte transduction

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Selective repopulation of normal mouse liver by hepatocytes transduced in vivo with recombinant adeno-associated virus

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A limited number of transducible hepatocytes restricts a wide-range linear vector dose response in recombinant adeno-associated virus-mediated liver transduction

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Active site mutant transgene confers tolerance to human beta-glucuronidase without affecting the phenotype of MPS VII mice.

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Induction of tolerance to human factor VIII in mice

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Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer

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Abnormal immune function in vivo in a murine model of lysosomal storage disease

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DNA determinants of structural and regulatory variation within the murine beta-glucuronidase gene complex

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10.1016/J.YMTHE.2004.05.029

1 reference

Europe PubMed Central

14 December 2017

1 reference

Europe PubMed Central

14 December 2017

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