Gene Therapy, An Issue of Hematology/Oncology Clinics of North America

Description

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.

Companion Products

• Gene Therapy, An Issue of Hematology/Oncology Clinics of North America

Table of Contents

Hematology/Oncology Clinics

Gene Therapy

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

Author Information

By Daniel E. Bauer, MD, PhD, Boston Children’s Hospital and Harvard Medical School and Donald B Kohn, MD, Professor, Departments of Microbiology Immunology and Molecular Genetics and Pediatrics, University of California (Los Angeles), Los Angeles CA, USA