(Q45422040)

English

Administration-route and gender-independent long-term therapeutic correction of phenylketonuria (PKU) in a mouse model by recombinant adeno-associated virus 8 pseudotyped vector-mediated gene transfer

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scholarly article

1 reference

Europe PubMed Central

14 December 2017

Administration-route and gender-independent long-term therapeutic correction of phenylketonuria (PKU) in a mouse model by recombinant adeno-associated virus 8 pseudotyped vector-mediated gene transfer (English)

1 reference

Europe PubMed Central

14 December 2017

vector-borne disease

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phenylketonuria

1 reference

based on heuristic

inferred from title

author name string

Ding Z

1

1 reference

Europe PubMed Central

14 December 2017

Georgiev P

2

1 reference

Europe PubMed Central

14 December 2017

Thöny B

3

1 reference

Europe PubMed Central

14 December 2017

language of work or name

1 reference

based on heuristic

inferred from title

publication date

1 April 2006

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Europe PubMed Central

14 December 2017

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Europe PubMed Central

14 December 2017

13

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Europe PubMed Central

14 December 2017

7

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Europe PubMed Central

14 December 2017

587-593

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Europe PubMed Central

14 December 2017

https://scigraph.springernature.com/pub.10.1038/sj.gt.3302684

0 references

PAHdb 2003: what a locus-specific knowledgebase can do.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302684

21 January 2018

The hyperphenylalaninemias of man and mouse

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302684

21 January 2018

National Institutes of Health Consensus Development Conference Statement: phenylketonuria: screening and management, October 16-18, 2000.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302684

21 January 2018

'Mommy, why can't I have a hamburger like the other kids?'.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302684

21 January 2018

Effect of stopping low-phenylalanine diet on intellectual progress of children with phenylketonuria

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302684

21 January 2018

Phenylketonuria in adulthood: a collaborative study

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302684

21 January 2018

Gene therapy with viral vectors

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302684

21 January 2018

Preclinical in vivo evaluation of pseudotyped adeno-associated virus vectors for liver gene therapy

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302684

21 January 2018

Total correction of hemophilia A mice with canine FVIII using an AAV 8 serotype.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302684

21 January 2018

Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302684

21 January 2018

Systemic delivery of genes to striated muscles using adeno-associated viral vectors.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302684

21 January 2018

Gene transfer into skeletal muscle using novel AAV serotypes.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302684

21 January 2018

Adenoviral and adeno-associated viral transfer of genes to the peripheral nervous system

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302684

21 January 2018

Transduction profiles of recombinant adeno-associated virus vectors derived from serotypes 2 and 5 in the nigrostriatal system of rats

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302684

21 January 2018

Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302684

21 January 2018

AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302684

21 January 2018

A phase II, double-blind, randomized, placebo-controlled clinical trial of tgAAVCF using maxillary sinus delivery in patients with cystic fibrosis with antrostomies.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302684

21 January 2018

Phase I trial of intranasal and endobronchial administration of a recombinant adeno-associated virus serotype 2 (rAAV2)-CFTR vector in adult cystic fibrosis patients: a two-part clinical study

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302684

21 January 2018

Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302684

21 January 2018

Immune responses to adenovirus and adeno-associated virus in humans.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302684

21 January 2018

A limited number of transducible hepatocytes restricts a wide-range linear vector dose response in recombinant adeno-associated virus-mediated liver transduction

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302684

21 January 2018

Unrestricted hepatocyte transduction with adeno-associated virus serotype 8 vectors in mice

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302684

21 January 2018

State-of-the-art 2003 on PKU gene therapy

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302684

21 January 2018

Long-term correction of hyperphenylalaninemia by AAV-mediated gene transfer leads to behavioral recovery in phenylketonuria mice

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302684

21 January 2018

Long-term enzymatic and phenotypic correction in the phenylketonuria mouse model by adeno-associated virus vector-mediated gene transfer

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302684

21 January 2018

Pahhph-5: a mouse mutant deficient in phenylalanine hydroxylase

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302684

21 January 2018

The phenylketonuria mouse model: a meeting review

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302684

21 January 2018

Tetrahydrobiopterin protects phenylalanine hydroxylase activity in vivo: implications for tetrahydrobiopterin-responsive hyperphenylalaninemia

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302684

21 January 2018

Influence of promoter and WHV post-transcriptional regulatory element on AAV-mediated transgene expression in the rat brain

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302684

21 January 2018

Reversal of hypopigmentation in phenylketonuria mice by adenovirus-mediated gene transfer

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302684

21 January 2018

Real-Time Quantitative Reverse Transcriptase-Polymerase Chain Reaction as a Method for Determining Lentiviral Vector Titers and Measuring Transgene Expression

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302684

21 January 2018

No evidence for tumorigenesis of AAV vectors in a large-scale study in mice.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302684

21 January 2018

Potential oncogene activity of the woodchuck hepatitis post-transcriptional regulatory element (WPRE).

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302684

21 January 2018

Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302684

21 January 2018

Liver gene therapy: advances and hurdles

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302684

21 January 2018

Gene therapy of liver diseases

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302684

21 January 2018

Metabolic engineering as therapy for inborn errors of metabolism--development of mice with phenylalanine hydroxylase expression in muscle

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302684

21 January 2018

Streamlined large-scale production of recombinant adeno-associated virus (rAAV) vectors

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302684

21 January 2018

Kupffer cells and not liver sinusoidal endothelial cells prevent lentiviral transduction of hepatocytes

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302684

21 January 2018

Impact of humoral immune response on distribution and efficacy of recombinant adeno-associated virus-derived acid alpha-glucosidase in a model of glycogen storage disease type II.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302684

21 January 2018

Characterization of mutations at the mouse phenylalanine hydroxylase locus

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302684

7 January 2021

based on heuristic

inferred from DOI database lookup

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10.1038/SJ.GT.3302684

1 reference

Europe PubMed Central

14 December 2017

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1 reference

Europe PubMed Central

14 December 2017

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