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Gene Therapy: The introduction of new genes into cells for the purpose of treating disease by restoring or adding gene expression. Techniques include insertion of retroviral vectors, transfection, homologous recombination, and injection of new genes into the nuclei of single cell embryos. The entire gene therapy process may consist of multiple steps. The new genes may be introduced into proliferating cells in vivo (e.g., bone marrow) or in vitro (e.g., fibroblast cultures) and the modified cells transferred to the site where the gene expression is required. Gene therapy may be particularly useful for treating enzyme deficiency diseases, hemoglobinopathies, and leukemias and may also prove useful in restoring drug sensitivity, particularly for leukemia.

RNA-based Reprogramming of Human Primary Fibroblasts into Induced Pluripotent Stem Cells

1Department of Dermatology, University of Colorado School of Medicine, Anschutz Medical Campus, 2Charles C. Gates Center for Regenerative Medicine, University of Colorado School of Medicine, Anschutz Medical Campus, 3Stem Cell Biobank and Disease Modeling Core, University of Colorado School of Medicine, Anschutz Medical Campus

JoVE 58687


 Developmental Biology

Analyzing Beneficial Effects of Nutritional Supplements on Intestinal Epithelial Barrier Functions During Experimental Colitis

1Department of Molecular Biomedicine, Center for Research and Advanced Studies of the National Polytechnic Institute, 2Department of Physiology, Biophysics and Neurosciences, Center for Research and Advanced Studies of the National Polytechnic Institute, 3Department of Infectomics and Molecular Pathogenesis, Center for Research and Advanced Studies of the National Polytechnic Institute

JoVE 55095


 Medicine

An Optimized Protocol to Analyze Glycolysis and Mitochondrial Respiration in Lymphocytes

1Laboratory of Mitochondrial Biology and Metabolism, National Heart, Lung, and Blood Institute, National Institutes of Health, 2Laboratory of Immunogenetics, National Institute of Allergy and Infectious Diseases, National Institutes of Health, 3Laboratory of Immunology, National Institute of Allergy and Infectious Diseases, National Institutes of Health

JoVE 54918


 Immunology and Infection

Antibody Binding Specificity for Kappa (Vκ) Light Chain-containing Human (IgM) Antibodies: Polysialic Acid (PSA) Attached to NCAM as a Case Study

1Department of Neurology, Mayo Clinic, 2Mayo Clinic Center for Multiple Sclerosis and Autoimmune Neurology, Mayo Clinic, 3Center for Regenerative Medicine, Neuroregeneration, Mayo Clinic, 4Division of Neonatal Medicine, Mayo Clinic, 5Department of Pediatric and Adolescent Medicine, Mayo Clinic

JoVE 54139


 Immunology and Infection

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