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.
Contains 14 relevant, practice-oriented topics including the evolution of gene therapy; viral vectors in hematopoietic stem cell gene therapy; gene editing in hematopoietic stem cells; nonintegrating vectors and engineered capsids; regulatory aspects of gene therapy; and more
Provides in-depth clinical reviews on gene-based therapies for pediatric blood diseases, offering actionable insights for clinical practice
Presents the latest information on this timely, focused topic under the leadership of experienced editors in the field. Authors synthesize and distill the latest research and practice guidelines to create clinically significant, topic-based reviews
1. Cover image - p. i 2. Title page - p. i 3. Table of Contents - p. i 4. Copyright - p. ii 5. Contributors - p. iii 6. Forthcoming Issues - p. xi 7. Preface - p. xiii 8. Evolution of Gene Therapy, Historical Perspective - p. 627 9. A Curative DNA Code for Hematopoietic Defects: Novel Cell Therapies for Monogenic Diseases of the Blood and Immune System - p. 647 10. Adeno-Associated Virus Vector Design–Moving the Adeno-Associated Virus to a Bioengineered Therapeutic Nanoparticle - p. 667 11. Regulatory Aspects of Gene Therapy: The Regulatory Life Cycle - p. 687 12. Chimeric Antigen Receptor T-cell Therapy: Current Status and Clinical Outcomes in Pediatric Hematologic Malignancies - p. 701 13. Genome-Edited T Cell Therapies - p. 729 14. Gene-Based Natural Killer Cell Therapies for the Treatment of Pediatric Hematologic Malignancies - p. 745 15. Gene Therapy for Hemoglobinopathies: Beta-Thalassemia, Sickle Cell Disease - p. 769 16. Hemophilia A/B - p. 797 17. Gene Therapy for Inborn Errors of Immunity: Severe Combined Immunodeficiencies - p. 813 18. Genes as Medicine: The Development of Gene Therapies for Inborn Errors of Immunity - p. 829 19. Gene Therapy for Pediatric Neurologic Disease - p. 853 20. Ex Vivo and In Vivo Gene Therapy for Mucopolysaccharidoses: State of the Art - p. 865
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.
Contains 14 relevant, practice-oriented topics including the evolution of gene therapy; viral vectors in hematopoietic stem cell gene therapy; gene editing in hematopoietic stem cells; nonintegrating vectors and engineered capsids; regulatory aspects of gene therapy; and more
Provides in-depth clinical reviews on gene-based therapies for pediatric blood diseases, offering actionable insights for clinical practice
Presents the latest information on this timely, focused topic under the leadership of experienced editors in the field. Authors synthesize and distill the latest research and practice guidelines to create clinically significant, topic-based reviews
1. Cover image - p. i 2. Title page - p. i 3. Table of Contents - p. i 4. Copyright - p. ii 5. Contributors - p. iii 6. Forthcoming Issues - p. xi 7. Preface - p. xiii 8. Evolution of Gene Therapy, Historical Perspective - p. 627 9. A Curative DNA Code for Hematopoietic Defects: Novel Cell Therapies for Monogenic Diseases of the Blood and Immune System - p. 647 10. Adeno-Associated Virus Vector Design–Moving the Adeno-Associated Virus to a Bioengineered Therapeutic Nanoparticle - p. 667 11. Regulatory Aspects of Gene Therapy: The Regulatory Life Cycle - p. 687 12. Chimeric Antigen Receptor T-cell Therapy: Current Status and Clinical Outcomes in Pediatric Hematologic Malignancies - p. 701 13. Genome-Edited T Cell Therapies - p. 729 14. Gene-Based Natural Killer Cell Therapies for the Treatment of Pediatric Hematologic Malignancies - p. 745 15. Gene Therapy for Hemoglobinopathies: Beta-Thalassemia, Sickle Cell Disease - p. 769 16. Hemophilia A/B - p. 797 17. Gene Therapy for Inborn Errors of Immunity: Severe Combined Immunodeficiencies - p. 813 18. Genes as Medicine: The Development of Gene Therapies for Inborn Errors of Immunity - p. 829 19. Gene Therapy for Pediatric Neurologic Disease - p. 853 20. Ex Vivo and In Vivo Gene Therapy for Mucopolysaccharidoses: State of the Art - p. 865
