This issue of Hematology/Oncology Clinics will focus on Gene Therapy. Topics include, but are not limited to Historical Perspective and Current Renaissance, Integrating Vectors, Nonintegrating Vectors, Gene Editing, Conditioning Therapies for Autologous HSCT, Approaches to Immunodeficiency, Approaches to Hemoglobinopathy, Approaches to Hemophilia, Hematopoietic Gene Therapies for Neurologic and Metabolic Disease, Gene Therapy Approaches to HIV and other Infectious Diseases, HSC Approaches to Cancer, and Gene Modified T Cell Therapies for Cancer.
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Preface: Gene Therapy
Historical Perspective on the Current Renaissance for Hematopoietic Stem Cell Gene Therapy
Integrating Vectors for Gene Therapy and Clonal Tracking of Engineered Hematopoiesis
Nonintegrating Gene Therapy Vectors
In Vivo Hematopoietic Stem Cell Transduction
Therapeutic Gene Editing Safety and Specificity
Gene Editing: Regulatory and Translation to Clinic
Opening Marrow Niches in Patients Undergoing Autologous Hematopoietic Stem Cell Gene Therapy
Gene Therapy Approaches to Immunodeficiency
Gene Therapy Approaches to Hemoglobinopathies
Gene Therapy for Hemophilia
Hematopoietic Gene Therapies for Metabolic and Neurologic Diseases
Gene Therapy Approaches to Human Immunodeficiency Virus and Other Infectious Diseases
Hematopoietic Stem Cell Approaches to Cancer
Gene Modified T Cell Therapies for Hematological Malignancies
Kohn, Donald B Professor, Departments of Microbiology Immunology and Molecular Genetics and Pediatrics, University of California (Los Angeles), Los Angeles CA, USA.