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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.

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


 Neuroscience

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


 Medicine

Gene Regulation and Targeted Therapy in Gastric Cancer Peritoneal Metastasis: Radiological Findings from Dual Energy CT and PET/CT

1Department of Radiology, Ruijin Hospital, Shanghai Jiao Tong University School of Medicine, 2Department of Nuclear Medicine, Ruijin Hospital, Shanghai Jiao Tong University School of Medicine, 3GE Healthcare China, 4Department of Surgery, Cedars-Sinai Medical Center

Video Coming Soon

JoVE 56526


 JoVE In-Press

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


 Immunology and Infection

Genome-Wide RNAi Screening to Identify Host Factors That Modulate Oncolytic Virus Therapy

1Children's Hospital of Eastern Ontario (CHEO) Research Institute, 2Department of Biology, Microbiology and Immunology, University of Ottawa, 3Department of Pediatrics, University of Ottawa, 4Department of Microbiology, Immunology and Infectious Diseases, Cumming School of Medicine, University of Calgary

Video Coming Soon

JoVE 56913


 JoVE In-Press

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

Bidirectional Retroviral Integration Site PCR Methodology and Quantitative Data Analysis Workflow

1UCLA AIDS Institute, University of California at Los Angeles (UCLA), 2Department of Microbiology, Immunology, & Molecular Genetics, University of California at Los Angeles (UCLA), 3Departments of Biomathematics and Mathematics, University of California at Los Angeles (UCLA), 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 (UCLA), 6Department of Veterinary Biosciences, College of Veterinary Medicine, The Ohio State University (OSU)

JoVE 55812


 Genetics

Whole Genome Sequencing of Candida Glabrata for Detection of Markers of Antifungal Drug Resistance

1Centre for Infectious Diseases and Microbiology-Public Health, Westmead Hospital, 2Centre for Infectious Diseases and Microbiology Laboratory Services, ICPMR, 3Marie Bashir Institute for Infectious Diseases and Biosecurity, The University of Sydney, 4Department of Microbiology and Infectious Diseases, Canberra Hospital and Health Services, Australian National University Medical School, 5Infection Management Services, Princess Alexandra Hospital, 6Department of Microbiology and Infectious Diseases, St. Vincent's Hospital, 7Department of Infectious Diseases, Peter MacCallum Cancer Centre

Video Coming Soon

JoVE 56714


 JoVE In-Press

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


 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


 Medicine

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

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


 Cancer Research

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

JoVE 55224


 Developmental Biology

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

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


 Cancer Research

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


 Immunology and Infection

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


 Bioengineering

Benefits of Cardiac Resynchronization Therapy in an Asynchronous Heart Failure Model Induced by Left Bundle Branch Ablation and Rapid Pacing

1Department of Cardiology, Shanghai Institute of Cardiovascular Diseases, Zhongshan Hospital, Fudan University, 2Department of Echocardiography, Shanghai Institute of Medical imaging, Shanghai Institute of Cardiovascular Diseases, Zhongshan Hospital, Fudan University, 3Department of Cardiac surgery, Shanghai Institute of Cardiovascular Diseases, Zhongshan Hospital, Fudan University

Video Coming Soon

JoVE 56439


 JoVE In-Press

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


 Neuroscience

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

JoVE 55827


 Cancer Research

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


 Immunology and Infection

Novel Diagnostics in Revision Arthroplasty: Implant Sonication and Multiplex Polymerase Chain Reaction

1Institute of Medical Microbiology, Immunology and Parasitology, University Hospital Bonn, 2Department of Orthopaedics and Trauma Surgery, University Hospital Bonn, 3Division of EU cooperation/Microbiology, Paul-Ehrlich-Institute

Video Coming Soon

JoVE 55147


 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

JoVE 55988


 Developmental Biology

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