Gene-Based Therapies for Pediatric Blood Diseases, An Issue of Hematology/Oncology Clinics of North America. The Clinics: Internal Medicine Volume 36-4

Table of Contents

Evolution of Gene Therapy, Historical PerspectiveA Curative DNA Code for Hematopoietic Defects: Novel Cell Therapies for Monogenic Diseases of the Blood and Immune SystemAdeno-Associated Virus Vector Design-Moving the Adeno-Associated Virus to a Bioengineered Therapeutic NanoparticleRegulatory Aspects of Gene Therapy: The Regulatory Life CycleChimeric Antigen Receptor T-cell Therapy: Current Status and Clinical Outcomes in Pediatric Hematologic MalignanciesGenome-Edited T Cell TherapiesGene-Based Natural Killer Cell Therapies for the Treatment of Pediatric Hematologic MalignanciesGene Therapy for Hemoglobinopathies: Beta-Thalassemia, Sickle Cell DiseaseHemophilia A/BGene Therapy for Inborn Errors of Immunity: Severe Combined ImmunodeficienciesGenes as Medicine: The Development of Gene Therapies for Inborn Errors of ImmunityGene Therapy for Pediatric Neurologic DiseaseEx Vivo and In Vivo Gene Therapy for Mucopolysaccharidoses: State of the Art