(Q35726773)

English

Preclinical evaluation of a clinical candidate AAV8 vector for ornithine transcarbamylase (OTC) deficiency reveals functional enzyme from each persisting vector genome

scientific article published on 7 November 2011

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scholarly article

1 reference

Europe PubMed Central

PubMed publication ID

9 August 2017

Preclinical evaluation of a clinical candidate AAV8 vector for ornithine transcarbamylase (OTC) deficiency reveals functional enzyme from each persisting vector genome (English)

1 reference

Europe PubMed Central

PubMed publication ID

9 August 2017

9

object named as

James M Wilson

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Europe PubMed Central

PubMed publication ID

9 August 2017

5

object named as

David Jones

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Europe PubMed Central

PubMed publication ID

9 August 2017

author name string

Lili Wang

1

1 reference

Europe PubMed Central

PubMed publication ID

9 August 2017

Hiroki Morizono

2

1 reference

Europe PubMed Central

PubMed publication ID

9 August 2017

Jianping Lin

3

1 reference

Europe PubMed Central

PubMed publication ID

9 August 2017

Peter Bell

4

1 reference

Europe PubMed Central

PubMed publication ID

9 August 2017

Deirdre McMenamin

6

1 reference

Europe PubMed Central

PubMed publication ID

9 August 2017

Hongwei Yu

7

1 reference

Europe PubMed Central

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9 August 2017

Mark L Batshaw

8

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Europe PubMed Central

PubMed publication ID

9 August 2017

publication date

7 November 2011

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Europe PubMed Central

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9 August 2017

Molecular Genetics and Metabolism

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Europe PubMed Central

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9 August 2017

105

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Europe PubMed Central

PubMed publication ID

9 August 2017

2

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Europe PubMed Central

PubMed publication ID

9 August 2017

203-211

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Europe PubMed Central

PubMed publication ID

9 August 2017

describes a project that uses

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Europe PubMed Central

11 June 2022

https://www.ebi.ac.uk/europepmc/webservices/rest/PMC3270700/fullTextXML

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Systemic errors in quantitative polymerase chain reaction titration of self-complementary adeno-associated viral vectors and improved alternative methods

1 reference

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1 reference

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Induction and prevention of severe hyperammonemia in the spfash mouse model of ornithine transcarbamylase deficiency using shRNA and rAAV-mediated gene delivery

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AAV2/8-mediated correction of OTC deficiency is robust in adult but not neonatal Spf(ash) mice

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Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primates

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Liver transplantation for inborn errors of metabolism

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Mammalian N-acetylglutamate synthase

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Alternative pathway therapy for urea cycle disorders: twenty years later

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Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors

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Adenovirus-mediated in vivo gene transfer rapidly protects ornithine transcarbamylase-deficient mice from an ammonium challenge

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

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Treatment of inborn errors of urea synthesis: activation of alternative pathways of waste nitrogen synthesis and excretion

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

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Treatment of urea cycle disorders

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Orthotopic liver transplantation for urea cycle enzyme deficiency

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Sustained correction of OTC deficiency in spf( ash) mice using optimized self-complementary AAV2/8 vectors.

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Systematic evaluation of AAV vectors for liver directed gene transfer in murine models.

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Long-term correction of ammonia metabolism and prolonged survival in ornithine transcarbamylase-deficient mice following liver-directed treatment with adeno-associated viral vectors.

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Developmental outcomes with early orthotopic liver transplantation for infants with neonatal-onset urea cycle defects and a female patient with late-onset ornithine transcarbamylase deficiency

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25 September 2018

Identifiers

10.1016/J.YMGME.2011.10.020

1 reference

Europe PubMed Central

PubMed publication ID

9 August 2017

PMC publication ID

1 reference

Europe PubMed Central

PubMed publication ID

9 August 2017

PubMed publication ID

1 reference

Europe PubMed Central

PubMed publication ID

9 August 2017

ResearchGate publication ID

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