Therapeutic neonatal hepatic gene therapy in mucopolysaccharidosis VII dogs (Q30476504)

13 September 2017

Therapeutic neonatal hepatic gene therapy in mucopolysaccharidosis VII dogs (English)

1 reference

13 September 2017

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11

Meg Sleeper

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13 September 2017

Katherine Parker Ponder

1

1 reference

13 September 2017

John R Melniczek

2

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13 September 2017

Lingfei Xu

3

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13 September 2017

Margaret A Weil

4

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13 September 2017

Thomas M O'Malley

5

1 reference

13 September 2017

Patricia A O'Donnell

6

1 reference

13 September 2017

Van W Knox

7

1 reference

13 September 2017

Gustavo D Aguirre

8

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13 September 2017

Hamutal Mazrier

9

1 reference

13 September 2017

N Matthew Ellinwood

10

1 reference

13 September 2017

Albert M Maguire

12

1 reference

13 September 2017

Susan W Volk

13

1 reference

13 September 2017

Robert L Mango

14

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13 September 2017

Jean Zweigle

15

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13 September 2017

John H Wolfe

16

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13 September 2017

Mark E Haskins

17

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13 September 2017

language of work or name

English

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publication date

13 September 2002

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Proceedings of the National Academy of Sciences of the United States of America

13 September 2017

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13 September 2017

99

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13 September 2017

20

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13 September 2017

13102-13107

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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

13 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=130593

Safety and efficacy of recombinant human alpha-galactosidase A replacement therapy in Fabry's disease

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=130593

Enzyme therapy for pompe disease with recombinant human alpha-glucosidase from rabbit milk

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=130593

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

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=130593

Systemic gene therapy for cardiovascular disease

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=130593

Long-term expression of human coagulation factor VIII and correction of hemophilia A after in vivo retroviral gene transfer in factor VIII-deficient mice

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=130593

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

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=130593

Prevalence of lysosomal storage disorders

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=130593

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

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=130593

Enzyme replacement therapy in a feline model of Maroteaux-Lamy syndrome

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=130593

Correction of lysosomal storage in the liver and spleen of MPS VII mice by implantation of genetically modified skin fibroblasts

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=130593

Enzyme replacement in a canine model of Hurler syndrome

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=130593

Phenotype correction in retinal pigment epithelium in murine mucopolysaccharidosis VII by adenovirus-mediated gene transfer

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=130593

Beta-glucuronidase deficiency in a dog: a model of human mucopolysaccharidosis VII.

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=130593

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

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=130593

Long-term effects of bone marrow transplantation in dogs with mucopolysaccharidosis I.

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=130593

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

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=130593

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

20 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=130593

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

20 June 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.192353499

Biodistribution, kinetics, and efficacy of highly phosphorylated and non-phosphorylated beta-glucuronidase in the murine model of mucopolysaccharidosis VII.

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.192353499

Enzyme replacement therapy in feline mucopolysaccharidosis I.

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.192353499

Enzyme-replacement therapy in mucopolysaccharidosis I.

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.192353499

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.192353499

Long-term follow-up following bone marrow transplantation for Hunter disease.

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.192353499

Long-term outcome of Hurler syndrome following bone marrow transplantation

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.192353499

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.192353499

Gene therapy for murine mucopolysaccharidosis type VII.

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.192353499

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.192353499

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.192353499

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.192353499

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.192353499

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.192353499

Disappearance of lysosomal storage in spleen and liver of mucopolysaccharidosis VII mice after transplantation of genetically modified bone marrow cells.

21 January 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=130593

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

26 November 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.192353499

Lipopolysaccharide potentiates the effect of hepatocyte growth factor on hepatocyte replication in rats by augmenting AP-1 activity

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.192353499

Induction of hepatocyte growth by intraportal infusion of HGF into beagle dogs

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.192353499

Effects of bone marrow transplantation on the cardiovascular abnormalities in canine mucopolysaccharidosis VII

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.192353499

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.192353499

Ocular abnormalities in the mucopolysaccharidoses after bone marrow transplantation. Longer follow-up

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.192353499

Newborn screening for lysosomal storage disorders

21 January 2018

1 reference

12 December 2020

Bone changes in mucopolysaccharidosis VI in cats and the effects of bone marrow transplantation: mechanical testing of long bones

1 reference

12 December 2020

Corneal endothelium in mucopolysaccharide storage disorders. Morphologic studies in animal models

1 reference

12 December 2020

Genetically corrected autologous stem cells engraft, but host immune responses limit their utility in canine alpha-L-iduronidase deficiency

1 reference

12 December 2020

Identifiers

DOI

10.1073/PNAS.192353499

1 reference

13 September 2017

0 references

1 reference

13 September 2017

PubMed publication ID

12232044

1 reference