In Vivo Transduction by Intravenous Injection of a Lentiviral Vector Expressing Human ADA into Neonatal ADA Gene Knockout Mice: A Novel Form of Enzyme Replacement Therapy for ADA Deficiency (Q63560628)

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English	In Vivo Transduction by Intravenous Injection of a Lentiviral Vector Expressing Human ADA into Neonatal ADA Gene Knockout Mice: A Novel Form of Enzyme Replacement Therapy for ADA Deficiency	article

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20 January 2020

In vivo transduction by intravenous injection of a lentiviral vector expressing human ADA into neonatal ADA gene knockout mice: a novel form of enzyme replacement therapy for ADA deficiency (English)

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20 January 2020

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Michael R. Blackburn

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20 January 2020

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20 January 2020

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Xiangyang Jin

2

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20 January 2020

Denise Petersen

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20 January 2020

Xingchao Wang

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20 January 2020

Fred Dorey

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20 January 2020

Ki Soo Kil

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20 January 2020

Melissa Aldrich

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20 January 2020

Denise A Carbonaro

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20 January 2020

Rodney E Kellems

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20 January 2020

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2 May 2006

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20 January 2020

Molecular Therapy

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20 January 2020

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20 January 2020

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20 January 2020

1110-1120

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20 January 2020

Complete sequence and structure of the gene for human adenosine deaminase

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7 January 2021

Hematopoietic stem-cell transplantation for the treatment of severe combined immunodeficiency.

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T lymphocyte ontogeny in adenosine deaminase-deficient severe combined immune deficiency after treatment with polyethylene glycol-modified adenosine deaminase

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7 January 2021

Long-term efficacy of enzyme replacement therapy for adenosine deaminase (ADA)-deficient severe combined immunodeficiency (SCID).

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7 January 2021

T lymphocyte-directed gene therapy for ADA- SCID: initial trial results after 4 years.

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7 January 2021

Gene therapy in peripheral blood lymphocytes and bone marrow for ADA- immunodeficient patients

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7 January 2021

T lymphocytes with a normal ADA gene accumulate after transplantation of transduced autologous umbilical cord blood CD34+ cells in ADA-deficient SCID neonates.

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7 January 2021

Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning

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7 January 2021

Disruption of the adenosine deaminase gene causes hepatocellular impairment and perinatal lethality in mice

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7 January 2021

Adenosine-deaminase-deficient mice die perinatally and exhibit liver-cell degeneration, atelectasis and small intestinal cell death

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7 January 2021

Tissue-specific rescue suggests that placental adenosine deaminase is important for fetal development in mice

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7 January 2021

Adenosine deaminase-deficient mice generated using a two-stage genetic engineering strategy exhibit a combined immunodeficiency

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7 January 2021

Metabolic consequences of adenosine deaminase deficiency in mice are associated with defects in alveogenesis, pulmonary inflammation, and airway obstruction

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7 January 2021

The use of enzyme therapy to regulate the metabolic and phenotypic consequences of adenosine deaminase deficiency in mice. Differential impact on pulmonary and immunologic abnormalities

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7 January 2021

The Moloney murine leukemia virus repressor binding site represses expression in murine and human hematopoietic stem cells

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Failure of SCID-X1 gene therapy in older patients

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Gene therapy of severe combined immunodeficiencies

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Biodistribution and toxicity studies of VSVG-pseudotyped lentiviral vector after intravenous administration in mice with the observation of in vivo transduction of bone marrow

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In vivo transduction of hematopoietic stem cells after neonatal intravenous injection of an amphotropic retroviral vector in mice

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7 January 2021

Neonatal gene therapy of MPS I mice by intravenous injection of a lentiviral vector

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

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Efficient hepatic delivery and expression from a recombinant adeno-associated virus 8 pseudotyped alpha1-antitrypsin vector

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

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Dynamic tracking of human hematopoietic stem cell engraftment using in vivo bioluminescence imaging

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Factors influencing the titer and infectivity of lentiviral vectors.

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Myeloproliferative virus, a cloned murine sarcoma virus with spleen focus-forming properties in adult mice

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Multiple modifications in cis elements of the long terminal repeat of retroviral vectors lead to increased expression and decreased DNA methylation in embryonic carcinoma cells.

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

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10.1016/J.YMTHE.2006.02.013

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20 January 2020

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Europe PubMed Central

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20 January 2020

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