Large animal models and gene therapy (Q36333340)

13 August 2017

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title

Large animal models and gene therapy (English)

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13 August 2017

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Margret Casal

1

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PubMed ID

16333317

13 August 2017

Mark Haskins

series ordinal

2

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13 August 2017

1 March 2006

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European Journal of Human Genetics

13 August 2017

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13 August 2017

14

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13 August 2017

3

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13 August 2017

266-272

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https://scigraph.springernature.com/pub.10.1038/sj.ejhg.5201535

13 August 2017

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cites work

A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

alpha-Galactosidase A deficient mice: a model of Fabry disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

Dramatically different phenotypes in mouse models of human Tay-Sachs and Sandhoff diseases

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

Simulation of hematopoiesis: implications for the gene therapy of lysosomal enzyme disorders

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

Behavior of hematopoietic stem cells in a large animal

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

Improved transduction of human sheep repopulating cells by retrovirus vectors pseudotyped with feline leukemia virus type C or RD114 envelopes

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

The sheep model of in utero gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

Update on the use of nonhuman primate models for preclinical testing of gene therapy approaches targeting hematopoietic cells.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

Online Mendelian Inheritance in Animals (OMIA): a comparative knowledgebase of genetic disorders and other familial traits in non-laboratory animals

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

IDID: inherited diseases in dogs: web-based information for canine inherited disease genetics

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

Semper fidelis: what man's best friend can teach us about human biology and disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

Canine X-linked severe combined immunodeficiency. A model for investigating the requirement for the common gamma chain (gamma c) in human lymphocyte development and function.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

Gene therapy for lysosomal storage diseases: the lessons and promise of animal models

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

Beta glucuronidase deficiency: report of clinical, radiologic, and biochemical features of a new mucopolysaccharidosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

Murine mucopolysaccharidosis type VII. Characterization of a mouse with beta-glucuronidase deficiency

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

Molecular basis of feline beta-glucuronidase deficiency: an animal model of mucopolysaccharidosis VII.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

Mucopolysaccharidosis VII in a cat.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

Cloning of the canine beta-glucuronidase cDNA, mutation identification in canine MPS VII, and retroviral vector-mediated correction of MPS VII cells.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

A single-base-pair deletion in the beta-glucuronidase gene accounts for the phenotype of murine mucopolysaccharidosis type VII

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

Neonatal gene transfer leads to widespread correction of pathology in a murine model of lysosomal storage disease.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

Transduction of hepatocytes after neonatal delivery of a Moloney murine leukemia virus based retroviral vector results in long-term expression of beta-glucuronidase in mucopolysaccharidosis VII dogs

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

Neonatal retroviral vector-mediated hepatic gene therapy reduces bone, joint, and cartilage disease in mucopolysaccharidosis VII mice and dogs

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

Therapeutic neonatal hepatic gene therapy in mucopolysaccharidosis VII dogs

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

Gene therapy ameliorates cardiovascular disease in dogs with mucopolysaccharidosis VII.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

Evaluation of pathological manifestations of disease in mucopolysaccharidosis VII mice after neonatal hepatic gene therapy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

A Line 1 insertion in the Factor IX gene segregates with mild hemophilia B in dogs

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

Canine hemophilia B resulting from a point mutation with unusual consequences

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

Systemic delivery of an adenoviral vector encoding canine factor VIII results in short-term phenotypic correction, inhibitor development, and biphasic liver toxicity in hemophilia A dogs.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

The Chapel Hill hemophilia A dog colony exhibits a factor VIII gene inversion

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

Muscle-directed gene transfer and transient immune suppression result in sustained partial correction of canine hemophilia B caused by a null mutation

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

AAV-mediated gene transfer for hemophilia

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

Influence of vector dose on factor IX-specific T and B cell responses in muscle-directed gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

Intravenous administration of an AAV-2 vector for the expression of factor IX in mice and a dog model of hemophilia B.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

Adeno-associated virus-mediated gene transfer for hemophilia B.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

Neonatal or hepatocyte growth factor-potentiated adult gene therapy with a retroviral vector results in therapeutic levels of canine factor IX for hemophilia B.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

The Briard dog: a new animal model of congenital stationary night blindness

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

Ultrastructural changes of the retina and the retinal pigment epithelium in Briard dogs with hereditary congenital night blindness and partial day blindness

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

Enhancing antimelanoma immune responses through apoptosis.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

Selective intraarterial gene delivery into a canine meningioma.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

In vivo tumor transfection with superantigen plus cytokine genes induces tumor regression and prolongs survival in dogs with malignant melanoma

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

Development of human granulocyte-macrophage colony-stimulating factor-transfected tumor cell vaccines for the treatment of spontaneous canine cancer

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

Intralesional lipid-complexed cytokine/superantigen immunogene therapy for spontaneous canine tumors

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

A comparative review of melanocytic neoplasms

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

Purification of feline lysosomal alpha-mannosidase, determination of its cDNA sequence and identification of a mutation causing alpha-mannosidosis in Persian cats

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

Bone marrow transplantation corrects the enzyme defect in neurons of the central nervous system in a lysosomal storage disease

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

Effective gene therapy for an inherited CNS disease in a large animal model

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

Treatment of experimental equine osteoarthritis by in vivo delivery of the equine interleukin-1 receptor antagonist gene

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

Gene therapy for arthritis

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

Comparative histopathology of grey-horse-melanoma and human malignant melanoma.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

Molecular definition of bovine argininosuccinate synthetase deficiency

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

Feline mucopolysaccharidosis VII due to beta-glucuronidase deficiency

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

7 January 2021

Histopathology, electrodiagnostic testing, and magnetic resonance imaging show significant peripheral and central nervous system myelin abnormalities in the cat model of alpha-mannosidosis

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

7 January 2021

Evaluation of gene therapy as a treatment for equine traumatic arthritis and osteoarthritis.

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

7 January 2021

Tumor regression induced by intratumoral injection of DNA coding for human interleukin 12 into melanoma metastases in gray horses

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201535

7 January 2021

Identifiers

DOI

10.1038/SJ.EJHG.5201535

1 reference

13 August 2017

1 reference

PubMed ID

16333317