(Q45888640)

17 December 2017

title

Developing adenoviral-mediated in vivo gene therapy for ornithine transcarbamylase deficiency (English)

1 reference

17 December 2017

0 references

1 reference

S E Raper

1

1 reference

17 December 2017

J M Wilson

2

1 reference

17 December 2017

M Yudkoff

3

1 reference

17 December 2017

M B Robinson

4

1 reference

17 December 2017

X Ye

5

1 reference

17 December 2017

M L Batshaw

6

1 reference

17 December 2017

publication date

1 January 1998

1 reference

17 December 2017

Journal of Inherited Metabolic Disease

published in

1 reference

17 December 2017

volume

21 Suppl 1

1 reference

17 December 2017

page(s)

119-137

1 reference

17 December 2017

The biochemical and molecular spectrum of ornithine transcarbamylase deficiency.

cites work

1 reference

12 December 2020

Genetic analysis of adenoviruses.

1 reference

12 December 2020

Risk of serious illness in heterozygotes for ornithine transcarbamylase deficiency

1 reference

12 December 2020

Efficient adenoviral-mediated ornithine transcarbamylase expression in deficient mouse and human hepatocytes

1 reference

12 December 2020

Ornithine Transcarbamylase Deficiency in Mutant Mice I. Studies on the Characterization of Enzyme Defect and Suitability as Animal Model of Human Disease

1 reference

12 December 2020

Biology of adenovirus vectors with E1 and E4 deletions for liver-directed gene therapy.

1 reference

12 December 2020

Hyperammonemia in women with a mutation at the ornithine carbamoyltransferase locus. A cause of postpartum coma

1 reference

12 December 2020

Adenovirus-mediated in vivo gene transfer and expression in normal rat liver

1 reference

12 December 2020

Alternative pathway therapy for urea cycle disorders

1 reference

12 December 2020

A pilot study of ex vivo gene therapy for homozygous familial hypercholesterolaemia

1 reference

12 December 2020

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

1 reference

12 December 2020

Evaluation of the transfer and expression in mice of an enzyme-encoding gene using a human adenovirus vector

1 reference

12 December 2020

Inactivation of E2a in recombinant adenoviruses improves the prospect for gene therapy in cystic fibrosis.

1 reference

12 December 2020

Orthotopic liver transplantation for urea cycle enzyme deficiency

1 reference

12 December 2020

Upregulation of class I major histocompatibility complex antigens by interferon gamma is necessary for T-cell-mediated elimination of recombinant adenovirus-infected hepatocytes in vivo

1 reference

12 December 2020

Retrospective survey of urea cycle disorders: Part 2. Neurological outcome in forty-nine Japanese patients with urea cycle enzymopathies

1 reference

12 December 2020

MHC class I-restricted cytotoxic T lymphocytes to viral antigens destroy hepatocytes in mice infected with E1-deleted recombinant adenoviruses.

1 reference

12 December 2020

Neurodevelopmental outcome of long-term therapy of urea cycle disorders in Japan

1 reference

12 December 2020

Adenovirus-mediated gene transfer into human hepatocytes: analysis of the biochemical functionality of transduced cells.

1 reference

12 December 2020

In vivo correction of low density lipoprotein receptor deficiency in the Watanabe heritable hyperlipidemic rabbit with recombinant adenoviruses.

1 reference

12 December 2020

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

1 reference

12 December 2020

Formulation and application of a numerical scoring system for assessing histological activity in asymptomatic chronic active hepatitis

1 reference

12 December 2020

Toxicity and response criteria of the Eastern Cooperative Oncology Group

1 reference

12 December 2020

Human ornithine transcarbamylase locus mapped to band Xp21.1 near the Duchenne muscular dystrophy locus

1 reference

12 December 2020

X-chromosome inactivation in human liver: confirmation of X-linkage of ornithine transcarbamylase

1 reference

12 December 2020

Safety and feasibility of liver-directed ex vivo gene therapy for homozygous familial hypercholesterolemia.

1 reference

12 December 2020

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

1 reference

12 December 2020

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

1 reference

12 December 2020

Transfer of the CFTR Gene to the Lung of Nonhuman Primates with E1-Deleted, E2a-Defective Recombinant Adenoviruses: A Preclinical Toxicology Study

1 reference

12 December 2020

Selective gene transfer into the liver of non-human primates with E1-deleted, E2A-defective, or E1-E4 deleted recombinant adenoviruses.

1 reference

12 December 2020

Late-onset ornithine transcarbamylase deficiency in male patients

1 reference

12 December 2020

Allopurinol-induced orotidinuria. A test for mutations at the ornithine carbamoyltransferase locus in women

1 reference

12 December 2020

Prolonged transgene expression in cotton rat lung with recombinant adenoviruses defective in E2a.

1 reference

12 December 2020

Cellular immunity to viral antigens limits E1-deleted adenoviruses for gene therapy

1 reference

12 December 2020

In vivo hepatic gene therapy: complete albeit transient correction of factor IX deficiency in hemophilia B dogs

1 reference

12 December 2020

Administration of an adenovirus containing the human CFTR cDNA to the respiratory tract of individuals with cystic fibrosis

1 reference

12 December 2020

Inborn errors of urea synthesis

1 reference

12 December 2020

Retrospective survey of urea cycle disorders: Part 1. Clinical and laboratory observations of thirty-two Japanese male patients with ornithine transcarbamylase deficiency

1 reference

12 December 2020

Liver transplantation for the treatment of urea cycle disorders

1 reference

12 December 2020

Estimated frequency of urea cycle enzymopathies in Japan

1 reference

12 December 2020

Ablation of E2A in recombinant adenoviruses improves transgene persistence and decreases inflammatory response in mouse liver

1 reference

12 December 2020

In vivo measurement of ureagenesis with stable isotopes

1 reference

12 December 2020

The sparse fur mouse as a model for gene therapy in ornithine carbamoyltransferase deficiency

1 reference

12 December 2020

Successful ex vivo gene therapy directed to liver in a patient with familial hypercholesterolaemia

1 reference

12 December 2020

Identifiers

DOI

10.1023/A:1005369926784

1 reference

17 December 2017

Dimensions Publication ID

1034273214

0 references

1 reference

17 December 2017