Stem cell selection in vivo using foamy vectors cures canine pyruvate kinase deficiency

Hematopoietic stem cell (HSC) gene therapy has cured immunodeficiencies including X-linked severe combined immunodeficiency (SCID-X1) and adenine deaminase deficiency (ADA). For these immunodeficiencies corrected cells have a selective advantage in vivo, and low numbers of gene-modified cells are su...

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Veröffentlicht in:	PloS one 2012-09, Vol.7 (9), p.e45173-e45173
Hauptverfasser:	Trobridge, Grant D, Beard, Brian C, Wu, Robert A, Ironside, Christina, Malik, Punam, Kiem, Hans-Peter
Format:	Artikel
Sprache:	eng
Schlagworte:	Acute Disease Adenine Adenine deaminase Analysis Anemia Anemia, Hemolytic, Congenital Nonspherocytic - enzymology Anemia, Hemolytic, Congenital Nonspherocytic - genetics Anemia, Hemolytic, Congenital Nonspherocytic - therapy Animals Biology Blood Bone marrow Cancer Cures Disease Disease Models, Animal DNA Modification Methylases - genetics DNA Modification Methylases - metabolism DNA Repair Enzymes - genetics DNA Repair Enzymes - metabolism Dogs Drug resistance Expression vectors Gene expression Gene therapy Genetic Therapy - methods Genetic Vectors Genomes Green Fluorescent Proteins - genetics Green Fluorescent Proteins - metabolism Hematopoietic Stem Cell Transplantation Hematopoietic stem cells Humans Immunodeficiency Kinases Medical research Medical treatment Medicine Methylguanine Methyltransferase Mutation Pyruvate kinase Pyruvate Kinase - deficiency Pyruvate Kinase - genetics Pyruvate Kinase - metabolism Pyruvate Metabolism, Inborn Errors - enzymology Pyruvate Metabolism, Inborn Errors - genetics Pyruvate Metabolism, Inborn Errors - therapy Pyruvic acid Rodents Severe combined immunodeficiency Spumavirus - genetics Stem cells Stem Cells - cytology Stem Cells - metabolism Transduction, Genetic Transgenes Transplantation Transplants & implants Treatment Outcome Tumor Suppressor Proteins - genetics Tumor Suppressor Proteins - metabolism Vectors (Biology) Viruses
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