Gene-Based Therapies for Pediatric Blood Diseases, An Issue of Hematology/Oncology Clinics of North America, 1st Edition
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.
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.
Key Features
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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.
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Provides in-depth clinical reviews on gene-based therapies for pediatric blood diseases, offering actionable insights for clinical practice.
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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.
Author Information
Edited by Nirali N. Shah, Lasker Clinical Research Scholar, Pediatric Oncology Branch, National Cancer Institute, Bethesda, Maryland, United States and Sung-Yun Pai, MD, Senior Investigator,Chief, Immune Deficiency Cellular Therapy Program,Center for Cancer Research, National Cancer Institute
