(Q45888388)

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Expression in blood cells may contribute to biochemical and pathological improvements after neonatal intravenous gene therapy for mucopolysaccharidosis VII in dogs

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Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/16275036

Expression in blood cells may contribute to biochemical and pathological improvements after neonatal intravenous gene therapy for mucopolysaccharidosis VII in dogs (English)

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/16275036

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author name string

Bin Wang

1

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/16275036

Thomas M O'Malley

2

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/16275036

Lingfei Xu

3

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/16275036

Charles Vite

4

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/16275036

Ping Wang

5

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/16275036

Patricia A O'Donnell

6

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/16275036

N Matthew Ellinwood

7

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/16275036

Mark E Haskins

8

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/16275036

Katherine Parker Ponder

9

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Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/16275036

publication date

7 November 2005

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Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/16275036

Molecular Genetics and Metabolism

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Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/16275036

87

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17 December 2017

http://europepmc.org/abstract/MED/16275036

1

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http://europepmc.org/abstract/MED/16275036

8-21

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Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/16275036

Incidence of the mucopolysaccharidoses in Western Australia

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2005.08.014

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2005.08.014

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2005.08.014

7 January 2021

Molecular basis of feline beta-glucuronidase deficiency: an animal model of mucopolysaccharidosis VII.

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2005.08.014

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2005.08.014

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2005.08.014

7 January 2021

Treatment of MPS VII (Sly disease) by allogeneic BMT in a female with homozygous A619V mutation

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2005.08.014

7 January 2021

Cord-blood transplants from unrelated donors in patients with Hurler's syndrome

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2005.08.014

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2005.08.014

7 January 2021

Enzyme replacement and enhancement therapies for lysosomal diseases

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2005.08.014

7 January 2021

Murine mucopolysaccharidosis type VII: long term therapeutic effects of enzyme replacement and enzyme replacement followed by bone marrow transplantation

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2005.08.014

7 January 2021

Enzyme-replacement therapy in mucopolysaccharidosis I.

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2005.08.014

7 January 2021

Gene therapy progress and prospects: gene therapy of lysosomal storage disorders

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2005.08.014

7 January 2021

Human CD34+ hematopoietic progenitor cell-directed lentiviral-mediated gene therapy in a xenotransplantation model of lysosomal storage disease.

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2005.08.014

7 January 2021

Autologous transplantation of retrovirally transduced bone marrow or neonatal blood cells into cats can lead to long-term engraftment in the absence of myeloablation

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2005.08.014

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2005.08.014

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2005.08.014

7 January 2021

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.

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2005.08.014

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2005.08.014

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2005.08.014

7 January 2021

Cloning of the canine beta-glucuronidase cDNA, mutation identification in canine MPS VII, and retroviral vector-mediated correction of MPS VII cells.

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2005.08.014

7 January 2021

Gene transfer of low levels of beta-glucuronidase corrects hepatic lysosomal storage in a large animal model of mucopolysaccharidosis VII.

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2005.08.014

7 January 2021

Therapeutic neonatal hepatic gene therapy in mucopolysaccharidosis VII dogs

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2005.08.014

7 January 2021

Neonatal retroviral vector-mediated hepatic gene therapy reduces bone, joint, and cartilage disease in mucopolysaccharidosis VII mice and dogs

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2005.08.014

7 January 2021

Gene therapy ameliorates cardiovascular disease in dogs with mucopolysaccharidosis VII.

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2005.08.014

7 January 2021

Transduction of hepatocytes after neonatal delivery of a Moloney murine leukemia virus based retroviral vector results in long-term expression of beta-glucuronidase in mucopolysaccharidosis VII dogs

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2005.08.014

7 January 2021

In vivo transduction of hematopoietic stem cells after neonatal intravenous injection of an amphotropic retroviral vector in mice

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2005.08.014

7 January 2021

The Moloney murine leukemia virus repressor binding site represses expression in murine and human hematopoietic stem cells

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2005.08.014

7 January 2021

Developmentally regulated mannose 6-phosphate receptor-mediated transport of a lysosomal enzyme across the blood-brain barrier

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2005.08.014

7 January 2021

Retrovirally transduced bone marrow has a therapeutic effect on brain in the mouse model of mucopolysaccharidosis IIIB.

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMGME.2005.08.014

7 January 2021

Identifiers

10.1016/J.YMGME.2005.08.014

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/16275036

1 reference

Europe PubMed Central

17 December 2017

http://europepmc.org/abstract/MED/16275036

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