Main description:

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.

Contents:

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/B
Gene 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