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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.

A Novel Surgical Approach for Intratracheal Administration of Bioactive Agents in a Fetal Mouse Model

1Molecular Virology and Gene Therapy, KU Leuven, 2Department of Woman and Child, KU Leuven, 3Neurobiology and Gene Therapy, KU Leuven, 4Division of Nuclear Medicine, KU Leuven, 5Biomedical NMR Unit/ MoSAIC, KU Leuven

JoVE 4219


 Medicine

Ultrahigh Resolution Mouse Optical Coherence Tomography to Aid Intraocular Injection in Retinal Gene Therapy Research

1Research Service, VA Western New York Healthcare System, 2Department of Ophthalmology, (Ross Eye Institute), Jacobs School of Medicine and Biomedical Sciences, University at Buffalo- SUNY, 3Pharmacology/Toxicology, Jacobs School of Medicine and Biomedical Sciences, University at Buffalo- SUNY, 4Physiology/Biophysics, Jacobs School of Medicine and Biomedical Sciences, University at Buffalo- SUNY, 5Neuroscience Program, Jacobs School of Medicine and Biomedical Sciences, University at Buffalo- SUNY, 6The RNA Institute, University at Buffalo- SUNY, 7The SUNY Eye Institute

JoVE 55894


 Medicine

CRISPR/Cas9-mediated Targeted Integration In Vivo Using a Homology-mediated End Joining-based Strategy

1Institute of Neuroscience, State Key Laboratory of Neuroscience, Key Laboratory of Primate Neurobiology, CAS Center for Excellence in Brain Science and Intelligence Technology, Shanghai Institutes for Biological Sciences, Chinese Academy of Sciences, 2College of Life Sciences, University of Chinese Academy of Sciences, 3School of Life Science and Technology, Shanghai Tech University, 4Institute of Biochemistry and Cell Biology, Shanghai Institutes for Biological Sciences, Chinese Academy of Sciences

JoVE 56844


 Genetics

Preparation and In Vitro Characterization of Magnetized miR-modified Endothelial Cells

1Reference and Translation Center for Cardiac Stem Cell Therapy (RTC), Department of Cardiac Surgery, University of Rostock, 2Physikalisch-Technische Bundesanstalt, 3Department of Radiology and Neuroradiology, Ernst-Moritz-Arndt-University Greifswald, 4Electron Microscopy Center, University of Rostock

JoVE 55567


 Medicine

Production of Pseudotyped Particles to Study Highly Pathogenic Coronaviruses in a Biosafety Level 2 Setting

1Department of Microbiology and Immunology, College of Veterinary Medicine, Cornell University, 2INRA, Virologie et Immunologie Moléculaires, 3Robert Frederick Smith School of Chemical and Biomolecular Engineering, Cornell University, 4Department of Microbiology, College of Agricultural and Life Sciences, Cornell University, 5Horae Gene Therapy Center, University of Massachusetts Medical School

Video Coming Soon

JoVE 59010


 JoVE In-Press

Isolation of Extracellular Vesicles from Murine Bronchoalveolar Lavage Fluid Using an Ultrafiltration Centrifugation Technique

1Department of Medicine, Division of Pulmonary and Critical Care, Women's Guild Lung Institute, Cedars-Sinai Medical Center, 2Department of Biomedical Sciences, Cedars-Sinai Medical Center, 3Department of Medicine, Smidt Heart Institute, Cedars-Sinai Medical Center

JoVE 58310


 Immunology and Infection

Generation of Cationic Nanoliposomes for the Efficient Delivery of In Vitro Transcribed Messenger RNA

1Department of Thoracic and Cardiovascular Surgery, Clinical Research Laboratory, University Medical Center, 2Atherothrombosis and Vascular Biology, Baker Heart & Diabetes Institute, 3Department of Medicine, Monash University

Video Coming Soon

JoVE 58444


 JoVE In-Press

Laser-assisted Lentiviral Gene Delivery to Mouse Fertilized Eggs

1Neurobiology Laboratory, National Institute of Environmental Health Sciences, 2Comparative Medicine Branch, National Institute of Environmental Health Sciences, 3Reproductive and Developmental Biology Laboratory, National Institute of Environmental Health Sciences, 4Signal Transduction Laboratory, National Institute of Environmental Health Sciences

JoVE 58327


 Biology

Lentiviral Vector-mediated Gene Therapy of Hepatocytes Ex Vivo for Autologous Transplantation in Swine

1Department of Surgery, Mayo Clinic, 2Midwest Fetal Care Center, Children's Hospitals and Clinics of Minnesota, 3Department of Surgery, Johns Hopkins, 4Pediatric Surgical Associates

JoVE 58399


 Medicine

Isolation, Characterization and MicroRNA-based Genetic Modification of Human Dental Follicle Stem Cells

1Reference and Translation Center for Cardiac Stem Cell Therapy (RTC), Department of Cardiac Surgery, Rostock University Medical Center, 2Department Life, Light and Matter of the Interdisciplinary Faculty at Rostock University, 3Department of Operative Dentistry and Periodontology, Rostock University Medical Center

JoVE 58089


 Genetics

Generation of Knock-out Primary and Expanded Human NK Cells Using Cas9 Ribonucleoproteins

1Center for Childhood Cancer and Blood Disease, Nationwide Children's Hospital, 2Nuffield Division of Clinical Laboratory Sciences, Radcliffe Department of Medicine, University of Oxford, 3Center for Clinical and Translational Research, Nationwide Children's Hospital Research Institute

JoVE 58237


 Immunology and Infection

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