(Q45875751)

17 December 2017

title

Early gestational gene transfer with targeted ATP7B expression in the liver improves phenotype in a murine model of Wilson's disease (English)

1 reference

17 December 2017

1 reference

J L Roybal

1

1 reference

17 December 2017

M Endo

2

1 reference

17 December 2017

A Radu

3

1 reference

17 December 2017

L Gray

4

1 reference

17 December 2017

C A Todorow

5

1 reference

17 December 2017

P W Zoltick

6

1 reference

17 December 2017

S Lutsenko

7

1 reference

17 December 2017

A W Flake

8

1 reference

17 December 2017

publication date

8 December 2011

1 reference

17 December 2017

published in

Gene Therapy

1 reference

17 December 2017

volume

19

1 reference

17 December 2017

issue

11

1 reference

17 December 2017

page(s)

1085-1094

1 reference

17 December 2017

Wilson disease: pathophysiology, diagnosis, treatment, and screening.

cites work

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.186

The Wilson's disease gene and phenotypic diversity

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.186

Clinical presentation, diagnosis and long-term outcome of Wilson's disease: a cohort study

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.186

Perspectives for gene therapy of Wilson disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.186

Chances and shortcomins of adenovirus-mediated ATP7B gene transfer in Wilson disease: proof of principle demonstrated in a pilot study with LEC rats.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.186

Restoration of copper metabolism and rescue of hepatic abnormalities in LEC rats, an animal model of Wilson disease, by expression of human ATP7B gene

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.186

The developmental stage determines the distribution and duration of gene expression after early intra-amniotic gene transfer using lentiviral vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.186

Early gestational gene transfer of IL-10 by systemic administration of lentiviral vector can prevent arthritis in a murine model

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.186

Robust in vivo transduction of nervous system and neural stem cells by early gestational intra amniotic gene transfer using lentiviral vector

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.186

Correction of ADAMTS13 deficiency by in utero gene transfer of lentiviral vector encoding ADAMTS13 genes

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.186

Efficient in vivo targeting of epidermal stem cells by early gestational intraamniotic injection of lentiviral vector driven by the keratin 5 promoter

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.186

Gene transfer to ocular stem cells by early gestational intraamniotic injection of lentiviral vector

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.186

Null mutation of the murine ATP7B (Wilson disease) gene results in intracellular copper accumulation and late-onset hepatic nodular transformation

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.186

Consequences of copper accumulation in the livers of the Atp7b-/- (Wilson disease gene) knockout mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.186

Atp7b-/- mice as a model for studies of Wilson's disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.186

Wilson disease: not just a copper disorder. Analysis of a Wilson disease model demonstrates the link between copper and lipid metabolism

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.186

High copper selectively alters lipid metabolism and cell cycle machinery in the mouse model of Wilson disease.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.186

Copper transport in mammalian cells: special care for a metal with special needs.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.186

Copper-induced trafficking of the cU-ATPases: a key mechanism for copper homeostasis

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.186

Wilson disease at a single cell level: intracellular copper trafficking activates compartment-specific responses in hepatocytes.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.186

Developmental landmarks in cardiac morphogenesis: Comparative chronology

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.186

A general method of in vitro preparation and specific mutagenesis of DNA fragments: study of protein and DNA interactions

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.186

Gene splicing and mutagenesis by PCR-driven overlap extension

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.186

Inhibiting HIV-1 infection in human T cells by lentiviral-mediated delivery of small interfering RNA against CCR5.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.186

Optimized large-scale production of high titer lentivirus vector pseudotypes

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.186

Early cell transplantation in LEC rats modeling Wilson's disease eliminates hepatic copper with reversal of liver disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.186

7 January 2021

Restoration of holoceruloplasmin synthesis in LEC rat after infusion of recombinant adenovirus bearing WND cDNA

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.186

7 January 2021

Lentiviral gene transfer ameliorates disease progression in Long-Evans cinnamon rats: an animal model for Wilson disease

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.186

7 January 2021

Stem cell and genetic therapies for the fetus

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.186

7 January 2021

Improvement of prenatal diagnosis of Wilson disease using microsatellite markers

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.186

7 January 2021

Development of a self-inactivating lentivirus vector.

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.186

7 January 2021

Increased probability of expression from modified retroviral vectors in embryonal stem cells and embryonal carcinoma cells

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.186

7 January 2021

Novel therapeutic approach for hemophilia using gene delivery of an engineered secreted activated Factor VII.

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.186

7 January 2021

Identifiers

DOI

10.1038/GT.2011.186

1 reference

17 December 2017

Dimensions Publication ID

1007885939

0 references

1 reference

17 December 2017