Transgene expression levels and kinetics determine risk of humoral immune response modeled in factor IX knockout and missense mutant mice

Immune responses leading to antibody-mediated elimination of the transgenic protein are a concern in gene replacement for congenital protein deficiencies, for which hemophilia is an important model. Although most hemophilia B patients have circulating non-functional but immunologically crossreactive...

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Veröffentlicht in:	Gene therapy 2007-03, Vol.14 (5), p.429-440
Hauptverfasser:	Zhang, T-P, Jin, D-Y, Wardrop, R M, Gui, T, Maile, R, Frelinger, J A, Stafford, D W, Monahan, P E
Format:	Artikel
Sprache:	eng
Schlagworte:	Adeno-associated virus Anesthesia. Intensive care medicine. Transfusions. Cell therapy and gene therapy Animal models Animals Antibodies Applied cell therapy and gene therapy Autoantibodies - immunology Biological and medical sciences Biomedical and Life Sciences Biomedicine Biotechnology Blood diseases Care and treatment Cell Biology Coagulation factors Cross Reactions Factor IX - genetics Factor IX - immunology Factor IX deficiency Fundamental and applied biological sciences. Psychology Gene Expression Gene Therapy Genetic aspects Genetic Therapy - adverse effects Genetic Therapy - methods Genetic Vectors - genetics Genotype & phenotype Genotypes Health. Pharmaceutical industry Hematologic and hematopoietic diseases Hemophilia Hemophilia A - blood Hemophilia A - immunology Hemophilia A - therapy Human Genetics Humans Immune response (humoral) Immunology Industrial applications and implications. Economical aspects Inhibitor drugs Kinetics Lymphocytes Medical sciences Methods Mice Mice, Knockout Mice, Mutant Strains Missense mutant Models, Biological Mutation Mutation, Missense Nanotechnology original-article Phenotypes Physiological aspects Platelet diseases and coagulopathies Proteins Prothrombin complex concentrate Risk Risk factors Rodents Serotypes Transduction, Genetic - methods Transfusions. Complications. Transfusion reactions. Cell and gene therapy Transgenes Transgenic animals
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