Genetically Corrected Autologous Stem Cells Engraft, But Host Immune Responses Limit Their Utility in Canine α-L-iduronidase Deficiency

Canine α-L-iduronidase (α-ID) deficiency, a model of the human storage disorder mucopolysaccharidosis type I (MPS I), is an ideal system in which to evaluate the clinical benefit of genetically corrected hematopoietic stem cells. We performed adoptive transfer of genetically corrected autologous hem...

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Veröffentlicht in:	Blood 1999-03, Vol.93 (6), p.1895-1905
Hauptverfasser:	Lutzko, Carolyn, Kruth, Stephen, Abrams-Ogg, Anthony C.G., Lau, Kathy, Li, Liheng, Clark, Brian R., Ruedy, Christine, Nanji, Shaherose, Foster, Robert, Kohn, Donald, Shull, Robert, Dubé, Ian D.
Format:	Artikel
Sprache:	eng
Schlagworte:	Animals Biological and medical sciences Biotechnology Bone Marrow Cells - enzymology Cells, Cultured Culture Media Dogs Fundamental and applied biological sciences. Psychology Gene Expression Gene therapy Gene Transfer Techniques Genetic Therapy Health. Pharmaceutical industry Hematopoietic Stem Cell Transplantation Hematopoietic Stem Cells - enzymology Hematopoietic Stem Cells - immunology Humans Iduronidase - deficiency Iduronidase - genetics Iduronidase - immunology Immunity Immunity, Cellular Industrial applications and implications. Economical aspects Lymphocyte Activation Mucopolysaccharidosis I - enzymology Mucopolysaccharidosis I - pathology Mucopolysaccharidosis I - therapy Polymerase Chain Reaction Retroviridae - genetics Transplantation, Autologous
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