(Q45628046)

English

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

scientific article published in June 2004

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scholarly article

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Europe PubMed Central

PubMed publication ID

14 December 2017

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 (English)

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Europe PubMed Central

PubMed publication ID

14 December 2017

vector-borne disease

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author name string

Seth D Hartung

1

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Europe PubMed Central

PubMed publication ID

14 December 2017

Joel L Frandsen

2

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Europe PubMed Central

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14 December 2017

Dao Pan

3

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14 December 2017

Brenda L Koniar

4

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14 December 2017

Patrick Graupman

5

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14 December 2017

Roland Gunther

6

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14 December 2017

Walter C Low

7

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14 December 2017

Chester B Whitley

8

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14 December 2017

R Scott McIvor

9

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14 December 2017

publication date

1 June 2004

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14 December 2017

Molecular Therapy

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14 December 2017

9

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14 December 2017

6

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14 December 2017

866-875

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Europe PubMed Central

PubMed publication ID

14 December 2017

Molecular genetics of mucopolysaccharidosis type I: diagnostic, clinical, and biological implications.

1 reference

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7 January 2021

Hurler and Hunter syndromes: mutual correction of the defect in cultured fibroblasts

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Long-term and high-dose trials of enzyme replacement therapy in the canine model of mucopolysaccharidosis I.

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Enzyme replacement therapy in feline mucopolysaccharidosis I.

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Reversal of clinical features of Hurler's disease and biochemical improvement after treatment by bone-marrow transplantation

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Long-term outcome of Hurler syndrome following bone marrow transplantation.

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Gene Transfer Strategies for Correction of Lysosomal Storage Disorders

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Gene therapy for mucopolysaccharidosis.

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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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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

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

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

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

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Therapeutic neonatal hepatic gene therapy in mucopolysaccharidosis VII dogs

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Enzyme replacement therapy for murine mucopolysaccharidosis type VII leads to improvements in behavior and auditory function

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Intracranial injection of recombinant adeno-associated virus improves cognitive function in a murine model of mucopolysaccharidosis type VII.

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Functional correction of established central nervous system deficits in an animal model of lysosomal storage disease with feline immunodeficiency virus-based vectors

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In vivo gene therapy of metachromatic leukodystrophy by lentiviral vectors: correction of neuropathology and protection against learning impairments in affected mice

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7 January 2021

Gene therapy for canine alpha-L-iduronidase deficiency: in utero adoptive transfer of genetically corrected hematopoietic progenitors results in engraftment but not amelioration of disease

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7 January 2021

In utero injection of alpha-L-iduronidase-carrying retrovirus in canine mucopolysaccharidosis type I: infection of multiple tissues and neonatal gene expression

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7 January 2021

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

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7 January 2021

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

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7 January 2021

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

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7 January 2021

Enzymatic correction and cross-correction of mucopolysaccharidosis type I fibroblasts by adeno-associated virus-mediated transduction of the alpha-L-iduronidase gene

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7 January 2021

Real-time quantitative polymerase chain reaction to assess gene transfer

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7 January 2021

Metabolic correction and cross-correction of mucopolysaccharidosis type II (Hunter syndrome) by retroviral-mediated gene transfer and expression of human iduronate-2-sulfatase

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7 January 2021

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

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7 January 2021

Enzyme replacement with recombinant beta-glucuronidase in murine mucopolysaccharidosis type VII: impact of therapy during the first six weeks of life on subsequent lysosomal storage, growth, and survival

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7 January 2021

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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7 January 2021

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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7 January 2021

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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Follow-up of nine patients with Hurler syndrome after bone marrow transplantation.

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7 January 2021

Light and electron microscopic features of the liver in mucopolysaccharidosis.

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7 January 2021

Coronary artery patency following long-term successful engraftment 14 years after bone marrow transplantation in the Hurler syndrome

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7 January 2021

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

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7 January 2021

Bone marrow transplantation in canine mucopolysaccharidosis I. Effects within the central nervous system

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Hurler syndrome: past, present, and future.

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Behavioral habituation to spatial novelty: interference and noninterference studies.

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Outcome of second hematopoietic cell transplantation in Hurler syndrome.

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Enzyme replacement and enhancement therapies: lessons from lysosomal disorders

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

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Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield

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

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10.1016/J.YMTHE.2004.03.011

1 reference

Europe PubMed Central

PubMed publication ID

14 December 2017

PubMed publication ID

1 reference

Europe PubMed Central

PubMed publication ID

14 December 2017

ResearchGate publication ID

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