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rdf:type |
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lifeskim:mentions |
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pubmed:issue |
7
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pubmed:dateCreated |
1999-4-7
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pubmed:abstractText |
Adenosine deaminase-deficient severe combined immunodeficiency was the first disease investigated for gene therapy because of a postulated production or survival advantage for gene-corrected T lymphocytes, which may overcome inefficient gene transfer. Four years after three newborns with this disease were given infusions of transduced autologous umbilical cord blood CD34+ cells, the frequency of gene-containing T lymphocytes has risen to 1-10%, whereas the frequencies of other hematopoietic and lymphoid cells containing the gene remain at 0.01-0.1%. Cessation of polyethylene glycol-conjugated adenosine deaminase enzyme replacement in one subject led to a decline in immune function, despite the persistence of gene-containing T lymphocytes. Thus, despite the long-term engraftment of transduced stem cells and selective accumulation of gene-containing T lymphocytes, improved gene transfer and expression will be needed to attain a therapeutic effect.
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pubmed:grant |
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pubmed:commentsCorrections |
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pubmed:language |
eng
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pubmed:journal |
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pubmed:citationSubset |
IM
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pubmed:chemical |
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pubmed:status |
MEDLINE
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pubmed:month |
Jul
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pubmed:issn |
1078-8956
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pubmed:author |
pubmed-author:AnnettGG,
pubmed-author:BarskyL WLW,
pubmed-author:BastianJJ,
pubmed-author:BlaeseR MRM,
pubmed-author:BowenTT,
pubmed-author:BrooksJJ,
pubmed-author:BurottoFF,
pubmed-author:CarbonaroDD,
pubmed-author:ChaoSS,
pubmed-author:CrooksGG,
pubmed-author:ElderMM,
pubmed-author:HershfieldM SMS,
pubmed-author:KapoorNN,
pubmed-author:KohnD BDB,
pubmed-author:MadsenEE,
pubmed-author:MuulLL,
pubmed-author:NolteJ EJE,
pubmed-author:ParkmanRR,
pubmed-author:ShigeokaAA,
pubmed-author:SmogorzewskaE MEM,
pubmed-author:SnyderF FFF,
pubmed-author:WardAA,
pubmed-author:WeinbergKK
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pubmed:issnType |
Print
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pubmed:volume |
4
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pubmed:owner |
NLM
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pubmed:authorsComplete |
Y
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pubmed:pagination |
775-80
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pubmed:dateRevised |
2010-12-28
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pubmed:meshHeading |
pubmed-meshheading:9662367-Adenosine Deaminase,
pubmed-meshheading:9662367-Animals,
pubmed-meshheading:9662367-Animals, Newborn,
pubmed-meshheading:9662367-Antigens, CD34,
pubmed-meshheading:9662367-Cell Line,
pubmed-meshheading:9662367-Flow Cytometry,
pubmed-meshheading:9662367-Gene Frequency,
pubmed-meshheading:9662367-Granulocytes,
pubmed-meshheading:9662367-Hematopoietic Stem Cell Transplantation,
pubmed-meshheading:9662367-Humans,
pubmed-meshheading:9662367-Leukocytes, Mononuclear,
pubmed-meshheading:9662367-Lymphocyte Count,
pubmed-meshheading:9662367-Mice,
pubmed-meshheading:9662367-Mice, SCID,
pubmed-meshheading:9662367-Polyethylene Glycols,
pubmed-meshheading:9662367-T-Lymphocytes,
pubmed-meshheading:9662367-Transformation, Genetic,
pubmed-meshheading:9662367-Transplantation, Autologous,
pubmed-meshheading:9662367-Transplantation Immunology,
pubmed-meshheading:9662367-Umbilical Cord
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pubmed:year |
1998
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pubmed:articleTitle |
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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pubmed:affiliation |
Division of Research Immunology/Bone Marrow Transplantation, Children's Hospital, Los Angeles, California 90027, USA.
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pubmed:publicationType |
Journal Article,
Research Support, U.S. Gov't, P.H.S.,
Research Support, Non-U.S. Gov't
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