Neonatal gene therapy of MPS I mice by intravenous injection of a lentiviral vector (Q40430846)

20 September 2017

Neonatal gene therapy of MPS I mice by intravenous injection of a lentiviral vector (English)

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20 September 2017

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20 September 2017

Hiroshi Kobayashi

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Denise Carbonaro

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Karen Pepper

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Denise Petersen

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Shundi Ge

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Holly Jackson

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20 September 2017

Hiroyuki Shimada

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20 September 2017

Rex Moats

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1 May 2005

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776-789

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Enzyme replacement with recombinant beta-glucuronidase in the newborn mucopolysaccharidosis type VII mouse

20 September 2017

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.10.006

Enzyme therapy for lysosomal storage disease: principles, practice, and prospects

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.10.006

Enzyme-replacement therapy in mucopolysaccharidosis I.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.10.006

Retrovirus-mediated transfer of the human alpha-L-iduronidase cDNA into human hematopoietic progenitor cells leads to correction in trans of Hurler fibroblasts

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.10.006

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.10.006

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.10.006

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

7 January 2021

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https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.10.006

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.10.006

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.10.006

Therapeutic neonatal hepatic gene therapy in mucopolysaccharidosis VII dogs

7 January 2021

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https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.10.006

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.10.006

Long-term normalization in the central nervous system, ocular manifestations, and skeletal deformities by a single systemic adenovirus injection into neonatal mice with mucopolysaccharidosis VII.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.10.006

Treatment of the mouse model of mucopolysaccharidosis I with retrovirally transduced bone marrow

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.10.006

Correction of metabolic, craniofacial, and neurologic abnormalities in MPS I mice treated at birth with adeno-associated virus vector transducing the human alpha-L-iduronidase gene

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.10.006

Activated microglia in cortex of mouse models of mucopolysaccharidoses I and IIIB

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.10.006

Dynamic tracking of human hematopoietic stem cell engraftment using in vivo bioluminescence imaging.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.10.006

Murine mucopolysaccharidosis type I: targeted disruption of the murine alpha-L-iduronidase gene

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.10.006

Interendothelial junctional changes underlie the developmental 'tightening' of the blood-brain barrier.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.10.006

Adenovirus-mediated gene transfer: influence of transgene, mouse strain and type of immune response on persistence of transgene expression

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.10.006

Efficiency of utilization of the simian virus 40 late polyadenylation site: effects of upstream sequences

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.10.006

Overexpression of the human lysosomal enzyme alpha-L-iduronidase in Chinese hamster ovary cells

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.10.006

Direct demonstration of binding of a lysosomal enzyme, alpha-L-iduronidase, to receptors on cultured fibroblasts

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.10.006

Simultaneous preparation and quantitation of proteoglycans by precipitation with alcian blue.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.10.006

A third-generation lentivirus vector with a conditional packaging system

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.10.006

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7 January 2021

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Identifiers

DOI

10.1016/J.YMTHE.2004.10.006

1 reference

20 September 2017

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