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

In this issue of Hematology/Oncology Clinics, guest editors Drs. Sung-Yun Pai and Nirali N. Shah bring their considerable expertise to the topic of Gene-Based Therapies for Pediatric Blood Diseases. Top experts in the field cover key topics such as CAR T-cell therapy: current status; engineered T cells; NK-cell therapy; hemoglobinopathies: beta-thalassemia, sickle cell disease; hemophilia A/B; primary immunodeficiencies; and more.

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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

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Elsevier
Publication Date
Jul 25, 2022

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