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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.
 JoVE Neuroscience

Limbal Approach-Subretinal Injection of Viral Vectors for Gene Therapy in Mice Retinal Pigment Epithelium

1Department of Biomedical Sciences, Seoul National University College of Medicine, 2FARB Laboratory, Biomedical Research Institute, Seoul National University Hospital, 3College of Life Sciences, Gwangju Institute of Science and Technology, 4Department of Ophthalmology, Seoul National University College of Medicine


JoVE 53030

 JoVE Medicine

Subretinal Injection of Gene Therapy Vectors and Stem Cells in the Perinatal Mouse Eye

1Bernard and Shirlee Brown Glaucoma Laboratory, Department of Ophthalmology, Columbia University, 2Institute of Human Nutrition, College of Physicians & Surgeons, Columbia University, 3Omics Laboratory, University of Iowa, 4Department of Ophthalmology and Visual Sciences, University of Iowa


JoVE 4286

 JoVE Immunology and Infection

Using the BLT Humanized Mouse as a Stem Cell based Gene Therapy Tumor Model

1Department of Medicine, Division of Hematology-Oncology, David Geffen School of Medicine at UCLA, 2UCLA AIDS Institute, 3Eli & Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA, 4Department of Medical and Molecular Pharmacology, David Geffen School of Medicine at UCLA, 5Department of Microbiology, Immunology and Molecular Genetics, David Geffen School of Medicine at UCLA


JoVE 4181

 JoVE In-Press

Bidirectional Retroviral Integration Site PCR Methodology and Quantitative Data Analysis Workflow

1UCLA AIDS Institute, University of California at Los Angeles, 2Department of Microbiology, Immunology, & Molecular Genetics, University of California at Los Angeles, 3Departments of Biomathematics and Mathematics, University of California at Los Angeles, 4Personalized Genomic Medicine Research Center, Division of Strategic Research Groups, Korea Research Institute of Bioscience and Biotechnology, 5Department of Medicine, University of California at Los Angeles, 6College of Veterinary Medicine, Department of Veterinary Biosciences, The Ohio State University

Video Coming Soon

JoVE 55812

 JoVE Medicine

An Affordable HIV-1 Drug Resistance Monitoring Method for Resource Limited Settings

1Africa Centre for Health and Population Studies, College of Health Sciences, University of KwaZulu-Natal, Durban, South Africa, 2Unit D11, Jembi Health Systems, 3Academic Medical Center, Department of Global Health, Amsterdam Institute for Global Health and Development (AIGHD), University of Amsterdam, 4Division of Infectious Diseases and Geographic Medicine, Centre for AIDS Research, Stanford Medical School


JoVE 51242

 JoVE Medicine

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

 JoVE Cancer Research

Establishment of Cancer Stem Cell Cultures from Human Conventional Osteosarcoma

1Department of Surgery and Translational Medicine (DCMT), University of Florence, 2Neurofarba Department, University of Florence, 3Department of Traumatology and General Orthopedics, Azienda Ospedaliera Universitaria Careggi


JoVE 53884

 JoVE In-Press

Isolation and Characterization of Mesenchymal Stromal Cells from Human Umbilical Cord and Fetal Placenta

1Department of Biological Sciences, Oakland University, 2OU-WB Institute for Stem Cell and Regenerative Medicine, 3Department of Obstetrics and Gynecology, St. John Provindence - Providence Park Hospital, 4Department of Neurosurgery, Beaumont Health System

Video Coming Soon

JoVE 55224

 JoVE Bioengineering

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

 JoVE Cancer Research

Next Generation Sequencing for the Detection of Actionable Mutations in Solid and Liquid Tumors

1Department of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, 2Division of Hematology/Oncology, Department of Medicine, Perelman School of Medicine, University of Pennsylvania, 3Abramson Cancer Center


JoVE 52758

 JoVE Immunology and Infection

Clinical Application of Sleeping Beauty and Artificial Antigen Presenting Cells to Genetically Modify T Cells from Peripheral and Umbilical Cord Blood

1Division of Pediatrics, U.T. MD Anderson Cancer Center, 2Department of Stem Cell Transplantation and Cellular Therapy, U.T. MD Anderson Cancer Center


JoVE 50070

 JoVE Bioengineering

Porous Silicon Microparticles for Delivery of siRNA Therapeutics

1Department of Nanomedicine, Houston Methodist Research Institute, 2MOE Key Laboratory of Bioinorganic and Synthetic Chemistry, School of Chemistry and Chemical Engineering, Sun Yat-sen University, 3Pediatrics Department of Union Hospital, Huazhong University of Science and Technology, 4CAS Key Laboratory for Biomedical Effects of Nanomaterials & Nanosafety, National Center for Nanoscience & Technology of China, 5Department of Medicine, Weill Cornell Medical College, 6Department of Cell and Developmental Biology, Weill Cornell Medical College


JoVE 52075

 JoVE Neuroscience

Modeling Astrocytoma Pathogenesis In Vitro and In Vivo Using Cortical Astrocytes or Neural Stem Cells from Conditional, Genetically Engineered Mice

1Department of Pathology and Laboratory Medicine, University of North Carolina School of Medicine, 2Lineberger Comprehensive Cancer Center, University of North Carolina School of Medicine, 3Division of Neuropathology, Department of Pathology and Laboratory Medicine, University of North Carolina School of Medicine, 4Curriculum in Genetics and Molecular Biology, University of North Carolina School of Medicine, 5Biological and Biomedical Sciences Program, University of North Carolina School of Medicine, 6Department of Radiation Oncology, Emory University School of Medicine, 7Department of Neurology, Neurosciences Center, University of North Carolina School of Medicine


JoVE 51763

 JoVE In-Press

A Unified Methodological Framework for Vestibular Schwannoma Research

1Eaton Peabody Laboratories, Department of Otolaryngology, Massachusetts Eye and Ear, 2Department of Otolaryngology, Harvard Medical School, 3Department of Otolaryngology, Vienna General Hospital, Medical University of Vienna, 4Program in Speech and Hearing Bioscience and Technology, Harvard Medical School

Video Coming Soon

JoVE 55827

 JoVE Immunology and Infection

Identifying DNA Mutations in Purified Hematopoietic Stem/Progenitor Cells

1Greehey Children's Cancer Research Institute, UT Health Science Center at San Antonio, 2Department of Cellular and Structural Biology, UT Health Science Center at San Antonio, 3Department of Pathology, UT Health Science Center at San Antonio, 4Department of Microbiology, UT Health Science Center at San Antonio, 5Cancer Therapy and Research Center, UT Health Science Center at San Antonio


JoVE 50752

 JoVE In-Press

Chondrogenic Pellet Formation from Cord Blood-derived Induced Pluripotent Stem Cells

1CiSTEM Laboratory, Convergent Research Consortium for Immunologic Disease, Division of Rheumatology, Seoul St. Mary's Hospital, College of Medicine, The Catholic University of Korea, 2Division of Rheumatology, Department of Internal Medicine, Seoul St. Mary's Hospital, Institute of Medical Science, College of Medicine, The Catholic University of Korea

Video Coming Soon

JoVE 55988

 JoVE Medicine

A Novel in vivo Gene Transfer Technique and in vitro Cell Based Assays for the Study of Bone Loss in Musculoskeletal Disorders

1Division of Rheumatology, Allergy and Clinical Immunology, University of California, Davis, 2Institute for Pediatric Regenerative Medicine, Shriners Hospitals for Children - Northern California, 3Department of Dermatology, University of California, Davis


JoVE 51810

 JoVE Cancer Research

Operating Procedures of the Electrochemotherapy for Treatment of Tumor in Dogs and Cats

1Clinic for Surgery and Small Animals, Veterinary Faculty, University of Ljubljana, 2Department of Experimental Oncology, Institute of Oncology Ljubljana, 3Faculty of Health Sciences, University of Primorska, 4IPBS (Institut de Pharmacologie et de Biologie Structurale), CNRS, 5IPBS (Institut de Pharmacologie et de Biologie Structurale), Université de Toulouse


JoVE 54760

 JoVE In-Press

Retroviral Scanning: Mapping MLV Integration Sites to Define Cell-specific Regulatory Regions

1Center for Genome Research, Department of Life Sciences, University of Modena and Reggio Emilia, 2Laboratory of chromatin and gene regulation during development, 3Institute for Biomedical Technologies, CNR, 4Généthon, 5Department of Life Sciences, University of Modena and Reggio Emilia, 6Paris Descartes, 7Sorbonne Paris Cité University, 8Imagine Institute

Video Coming Soon

JoVE 55919

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