About: 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

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About: 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 Goto Sponge NotDistinct Permalink

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rdf:type	Item
description	article (en) wetenschappelijk artikel (nl) наукова стаття, опублікована в червні 2006 (uk) im Mai 2006 veröffentlichter wissenschaftlicher Artikel (de)
publication date	wds:Q63560628-FBF84A00-FFE0-4BC5-A4A5-BFAA4B7C0F77
publication date	2006-05-02 00:00:00Z (xsd:dateTime)
cites work	wds:Q63560628-03120C82-970B-4EAC-849B-FDFEC68030DF wds:Q63560628-08CC33F0-DD44-4DF8-A7FA-FFDE376563A6 wds:Q63560628-0DBDB114-BF3E-4B60-A713-D4E0BBA54992 wds:Q63560628-187E8856-CE9B-43E6-839F-FF6E9D933914 wds:Q63560628-3AE4E41A-D4D7-4B98-8E33-BA9DA41B9BCD wds:Q63560628-3CA1F85E-D37C-4462-BC1C-31E6F46B5C32 wds:Q63560628-4C222653-3FF7-426F-BDD6-A5060DD0DCF9 wds:Q63560628-5768369D-2DAC-4027-8151-7E3D83BFC038 wds:Q63560628-5ABF8566-F38A-4DF8-B2C3-530567C38C62 wds:Q63560628-6374B89A-E443-45E9-82CE-DFFD9229BC91 wds:Q63560628-6C88A1F5-7A53-4A23-B475-EBD28BFBE343 wds:Q63560628-72A4F95F-E866-4751-B390-29B5C08FC039 wds:Q63560628-73DE7490-04E8-469B-8684-142E858B3E0A wds:Q63560628-823CBA87-03CB-4868-8021-7CA49C57D901 wds:Q63560628-8A390C19-4FE4-435A-94E0-D759BDF4ED54 wds:Q63560628-9749F3BB-12F8-45CD-95F9-B3769126F8DC wds:Q63560628-98986FEC-F5DA-4D19-9D97-8272D4D38AF8 wds:Q63560628-98E9F1A3-66B9-44BF-BE5A-0F2D783AD624 wds:Q63560628-A4D41887-052A-4373-A859-1638BAF04F33 wds:Q63560628-AE0A6479-B2B0-4DC2-A532-1CF870C984DA wds:Q63560628-B3FE9F19-CD9F-4E5D-A590-0792F9951897 wds:Q63560628-BF72360F-F055-464F-B675-C9CFD32CC6D4 wds:Q63560628-C1F7F7B7-48C5-4D99-ABB9-4E5281CA2DAD wds:Q63560628-CCA0D0ED-BED9-4F05-A7E3-191F7DEB4D52 wds:Q63560628-DF2B45D3-2B9A-4E22-AEE0-5FD16D4F8E73 wds:Q63560628-E99A0B01-FA4B-46D1-B0CB-D8A079F0C94E wds:Q63560628-F3A706B4-3DB9-4CAA-AB7E-BC94B785CD7E wds:Q63560628-FB8B8EC3-BEE8-4FDC-8452-04B391B23BA8
cites work	Complete sequence and structure of the gene for human adenosine deaminase T lymphocyte ontogeny in adenosine deaminase-deficient severe combined immune deficiency after treatment with polyethylene glycol-modified adenosine deaminase Unrestricted hepatocyte transduction with adeno-associated virus serotype 8 vectors in mice Adenosine deaminase-deficient mice generated using a two-stage genetic engineering strategy exhibit a combined immunodeficiency Adenosine-deaminase-deficient mice die perinatally and exhibit liver-cell degeneration, atelectasis and small intestinal cell death Metabolic consequences of adenosine deaminase deficiency in mice are associated with defects in alveogenesis, pulmonary inflammation, and airway obstruction Disruption of the adenosine deaminase gene causes hepatocellular impairment and perinatal lethality in mice Neonatal gene transfer leads to widespread correction of pathology in a murine model of lysosomal storage disease. 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 T lymphocytes with a normal ADA gene accumulate after transplantation of transduced autologous umbilical cord blood CD34+ cells in ADA-deficient SCID neonates. Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning Hematopoietic stem-cell transplantation for the treatment of severe combined immunodeficiency. Gene therapy of severe combined immunodeficiencies Tissue-specific rescue suggests that placental adenosine deaminase is important for fetal development in mice Gene therapy in peripheral blood lymphocytes and bone marrow for ADA- immunodeficient patients T lymphocyte-directed gene therapy for ADA- SCID: initial trial results after 4 years. Efficient hepatic delivery and expression from a recombinant adeno-associated virus 8 pseudotyped alpha1-antitrypsin vector Long-term efficacy of enzyme replacement therapy for adenosine deaminase (ADA)-deficient severe combined immunodeficiency (SCID). Biodistribution and toxicity studies of VSVG-pseudotyped lentiviral vector after intravenous administration in mice with the observation of in vivo transduction of bone marrow Factors influencing the titer and infectivity of lentiviral vectors. Dynamic tracking of human hematopoietic stem cell engraftment using in vivo bioluminescence imaging. Failure of SCID-X1 gene therapy in older patients Neonatal gene therapy of MPS I mice by intravenous injection of a lentiviral vector Increased probability of expression from modified retroviral vectors in embryonal stem cells and embryonal carcinoma cells Myeloproliferative virus, a cloned murine sarcoma virus with spleen focus-forming properties in adult mice 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. In vivo transduction of hematopoietic stem cells after neonatal intravenous injection of an amphotropic retroviral vector in mice The Moloney murine leukemia virus repressor binding site represses expression in murine and human hematopoietic stem cells
author name string	wds:Q63560628-289EDB98-3460-4956-BC41-3BC8BEA23345 wds:Q63560628-55D77A9B-61E1-4A85-8E97-FDB07681A26A wds:Q63560628-66FA4D6C-3D40-4AAB-B5A4-FB23661F4CC5 wds:Q63560628-8CD951B9-0BEE-4706-BE77-19841610EC75 wds:Q63560628-8EA0A0DA-AA9A-42EC-AA6D-8E378EB1B29B wds:Q63560628-BA13F64E-DEAE-4D75-881D-F01623593883 wds:Q63560628-CF6DEEBF-5E65-4FA1-8EF0-2A98439FCC2B wds:Q63560628-F1760CD3-E8DA-485E-AF48-16C3FB3790E2
author name string	Melissa Aldrich Rodney E Kellems Denise A Carbonaro Fred Dorey Xingchao Wang Denise Petersen Xiangyang Jin Ki Soo Kil
rdfs:label	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 (en) 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 (nl)
skos:prefLabel	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 (en) 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 (nl)
name	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 (en) 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 (nl)
author	wds:Q63560628-2347E56C-BDA6-49C2-ABCB-4FB681DD296A wds:Q63560628-81045F81-2DEE-47A9-831E-B91B592ED611
author	Michael R. Blackburn Donald B. Kohn
title	wds:Q63560628-81577D73-1510-4143-A3B5-1FD52ABB5712
title	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 (en)
page(s)	wds:Q63560628-75170A01-FDEC-4764-A236-863B0BA255B5
page(s)	1110-1120
instance of	wds:Q63560628-627FE1D5-E04B-43D9-A93E-73C8F367B963
instance of	scholarly article
main subject	wds:Q63560628-0ED8F39A-464A-4CE3-9515-135E26FC38C2
main subject	drug discovery
PubMed ID	wds:Q63560628-5E5A867E-B1A4-4BAD-AAD8-62C244EDFA6B
PubMed ID	http://rdf.ncbi.nlm.nih.gov/pubchem/reference/16651028
PubMed ID	16651028

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