Gene-Based Therapies for Pediatric Blood Diseases, An Issue of Hematology/Oncology Clinics of North America

Evolution of Gene Therapy, Historical Perspective
A Curative DNA Code for Hematopoietic Defects: Novel Cell Therapies for Monogenic Diseases of the Blood and Immune System
Adeno-Associated Virus Vector Design–Moving the Adeno-Associated Virus to a Bioengineered Therapeutic Nanoparticle
Regulatory Aspects of Gene Therapy: The Regulatory Life Cycle
Chimeric Antigen Receptor T-cell Therapy: Current Status and Clinical Outcomes in Pediatric Hematologic Malignancies
Genome-Edited T Cell Therapies
Gene-Based Natural Killer Cell Therapies for the Treatment of Pediatric Hematologic Malignancies
Gene Therapy for Hemoglobinopathies: Beta-Thalassemia, Sickle Cell Disease
Hemophilia A/BGene Therapy for Inborn Errors of Immunity: Severe Combined Immunodeficiencies
Genes as Medicine: The Development of Gene Therapies for Inborn Errors of Immunity
Gene Therapy for Pediatric Neurologic Disease
Ex Vivo and In Vivo Gene Therapy for Mucopolysaccharidoses: State of the Art