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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.

The Aortic Ring Co-culture Assay: A Convenient Tool to Assess the Angiogenic Potential of Mesenchymal Stromal Cells In Vitro

1Create Fertility Centre, 2Department of Physiology, University of Toronto, 3Department of Obstetrics and Gynecology, University of Toronto, 4Department of Medical Sciences, University of Toronto, 5Department of Obstetrics and Gynecology, Women's College Hospital

JoVE 56083


 Developmental Biology

Preclinical Model of Hind Limb Ischemia in Diabetic Rabbits

1Department of Biomedical Engineering, University of Texas at Austin, 2Division of Cardiology, University of Texas McGovern Medical School, 3Center for Laboratory Animal Medicine and Care, UT Health Science Center at Houston, 4Memorial Herman Heart and Vascular Center, Texas Medical Center, 5Institute for Cellular and Molecular Biology, University of Texas at Austin, 6The Institute for Computational Engineering and Sciences, University of Texas at Austin, 7Institute for Biomaterials, Drug Delivery and Regenerative Medicine, University of Texas at Austin

JoVE 58964


 Medicine

Lentiviral Vector Platform for the Efficient Delivery of Epigenome-editing Tools into Human Induced Pluripotent Stem Cell-derived Disease Models

1Department of Neurology, Duke University Medical Center, 2Center for Genomic and Computational Biology, Duke University Medical Center, 3Viral Vector Core, Duke University Medical Center, 4Division of Gynecologic Oncology, Department of Obstetrics and Gynecology, Duke University Medical Center

JoVE 59241


 Genetics

Construction of Defined Human Engineered Cardiac Tissues to Study Mechanisms of Cardiac Cell Therapy

1Cardiovascular Research Center, Icahn School of Medicine at Mount Sinai, 2The Mindich Child Health and Development Institute, Icahn School of Medicine at Mount Sinai, 3Stem Cell & Regenerative Medicine Consortium, LKS Faculty of Medicine, University of Hong Kong

JoVE 53447


 Bioengineering

Generation of Integration-free Induced Pluripotent Stem Cells from Human Peripheral Blood Mononuclear Cells Using Episomal Vectors

1State Key Laboratory of Experimental Hematology, Institute of Hematology and Blood Disease Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, 2Division of Regenerative Medicine, Department of Medicine, Loma Linda University, 3Department of Orthopaedic Surgery, Loma Linda University, 4Center for Stem Cell Medicine, Chinese Academy of Medical Sciences, 5Department of Stem Cell & Regenerative Medicine, Peking Union Medical College, 6Collaborative Innovation Center for Cancer Medicine, 7Tianjin Key Laboratory of Blood Cell Therapy and Technology

JoVE 55091


 Developmental Biology

Processing of Bronchoalveolar Lavage Fluid and Matched Blood for Alveolar Macrophage and CD4+ T-cell Immunophenotyping and HIV Reservoir Assessment

1Research Institute of McGill University Health Centre, 2Department of Biological Sciences, Université de Québec à Montréal, 3Department of Microbiology & Immunology, McGill University, 4Department of Medicine, Division of Infectious Diseases, McGill University, 5Centre de Recherche du Centre Hospitalier de l'Université de Montréal, 6Département de microbiologie, infectiologie et immunologie, Université de Montréal

Video Coming Soon

JoVE 59427


 JoVE In-Press

Immunoglobulin Gene Sequence Analysis In Chronic Lymphocytic Leukemia: From Patient Material To Sequence Interpretation

1Institute of Applied Biosciences, Centre for Research and Technology Hellas, 2Department of Immunology, Genetics and Pathology, Science for Life Laboratory, Uppsala University, 3Department of Molecular Medicine and Surgery, Karolinska Institutet, 4Division of Immunology, Transplantation and Infectious, IRCCS San Raffaele Scientific Institute, 5Department of Immunology, Laboratory for Medical Immunology, Erasmus University Medical Center, 6Hematology Department, Nikea General Hospital, 7Assistance publique - Hôpitaux de Paris (AP-HP), Hopital Pitié-Salpêtrière, Department of Hematology, and UPMC University Paris 06, UMRS 1138, 8Division of Experimental Oncology, IRCCS Istituto Scientifico San Raffaele and Università Vita-Salute San Raffaele

JoVE 57787


 Cancer Research

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