(Q45036081)

8 December 2017

Long-term correction of ornithine transcarbamylase deficiency by WPRE-mediated overexpression using a helper-dependent adenovirus (English)

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8 December 2017

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1 reference

Asad Mian

1

1 reference

8 December 2017

W Michael McCormack

2

1 reference

8 December 2017

Viraj Mane

3

1 reference

8 December 2017

Soledad Kleppe

4

1 reference

8 December 2017

Philip Ng

5

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8 December 2017

Milton Finegold

6

1 reference

8 December 2017

William E O'Brien

7

1 reference

8 December 2017

John R Rodgers

8

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8 December 2017

Arthur L Beaudet

9

1 reference

8 December 2017

Brendan Lee

10

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8 December 2017

1 September 2004

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8 December 2017

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8 December 2017

10

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8 December 2017

3

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8 December 2017

492-499

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Neurologic outcome in children with inborn errors of urea synthesis. Outcome of urea-cycle enzymopathies.

8 December 2017

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.05.036

Neonatal onset ornithine transcarbamylase deficiency: A retrospective analysis

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.05.036

Long-term outcome of patients with urea cycle disorders and the question of neonatal screening

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.05.036

Urea-cycle disorders as a paradigm for inborn errors of hepatocyte metabolism

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.05.036

Long-term correction of urea cycle disorders

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.05.036

Liver transplantation in urea cycle disorders

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.05.036

An integrated approach to the diagnosis and prospective management of partial ornithine transcarbamylase deficiency

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.05.036

Hepatocyte gene therapy in a large animal: a neonatal bovine model of citrullinemia

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.05.036

Prolonged metabolic correction in adult ornithine transcarbamylase-deficient mice with adenoviral vectors

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.05.036

Patient selection may affect gene therapy success. Dominant negative effects observed for ornithine transcarbamylase in mouse and human hepatocytes

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.05.036

Correction of ornithine transcarbamylase deficiency in adult spf(ash) mice and in OTC-deficient human hepatocytes with recombinant adenoviruses bearing the CAG promoter

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.05.036

Differences in the human and mouse amino-terminal leader peptides of ornithine transcarbamylase affect mitochondrial import and efficacy of adenoviral vectors

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.05.036

Improvements in gene therapy: averting the immune response to adenoviral vectors

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.05.036

Differential activation of innate immune responses by adenovirus and adeno-associated virus vectors

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.05.036

Activation of innate immunity in nonhuman primates following intraportal administration of adenoviral vectors.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.05.036

Acute cytokine response to systemic adenoviral vectors in mice is mediated by dendritic cells and macrophages

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.05.036

Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.05.036

Use of a liver-specific promoter reduces immune response to the transgene in adenoviral vectors

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.05.036

Efficacy of gene therapy for a prototypical lysosomal storage disease (GSD-II) is critically dependent on vector dose, transgene promoter, and the tissues targeted for vector transduction

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.05.036

Effect of promoters and enhancers on expression, transgene DNA persistence, and hepatotoxicity after adenoviral gene transfer of human apolipoprotein A-I

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.05.036

Separating fact from fiction: assessing the potential of modified adenovirus vectors for use in human gene therapy

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.05.036

Progress toward gene therapy of Duchenne muscular dystrophy

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.05.036

Enhanced expression of transgenes from adeno-associated virus vectors with the woodchuck hepatitis virus posttranscriptional regulatory element: implications for gene therapy

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.05.036

CRM1-dependent function of a cis-acting RNA export element

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.05.036

Tissue-specific, developmental, hormonal, and dietary regulation of rat phosphoenolpyruvate carboxykinase-human growth hormone fusion genes in transgenic mice

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.05.036

Optimization of the helper-dependent adenovirus system for production and potency in vivo

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.05.036

Diagnostic value of orotic acid excretion in heritable disorders of the urea cycle and in hyperammonemia due to organic acidurias

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.05.036

Improved system for helper-dependent adenoviral vector production

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.05.036

Acute toxicity after high-dose systemic injection of helper-dependent adenoviral vectors into nonhuman primates.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.05.036

Production and characterization of human 293 cell lines expressing the site-specific recombinase Cre.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.05.036

A helper-dependent adenovirus vector system: removal of helper virus by Cre-mediated excision of the viral packaging signal

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.05.036

Woodchuck hepatitis virus contains a tripartite posttranscriptional regulatory element.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.05.036

Woodchuck hepatitis virus posttranscriptional regulatory element enhances expression of transgenes delivered by retroviral vectors.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.05.036

inferred from DOI database lookup

7 January 2021

based on heuristic

Role of viral antigens in destructive cellular immune responses to adenovirus vector-transduced cells in mouse lungs.

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2004.05.036

inferred from DOI database lookup

7 January 2021

based on heuristic

Identifiers

DOI

10.1016/J.YMTHE.2004.05.036

1 reference

8 December 2017

1 reference