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

1st Edition, Volume 36-4 - July 19, 2022
Editors: Nirali N. Shah, Sung-Yun Pai
Language: English
Hardback ISBN:
9 7 8 - 0 - 3 2 3 - 9 8 7 7 5 - 2
eBook ISBN:
9 7 8 - 0 - 3 2 3 - 9 8 7 7 6 - 9

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 fo… Read more

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

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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.

Cover image
Title page
Table of Contents
Copyright
Contributors
Forthcoming Issues
Preface
Evolution of Gene Therapy, Historical Perspective
Key points
Introduction to theoretic concepts and early background history impacting hematopoietic stem cell gene therapy
Design of integrating vectors used for hematopoietic stem cells gene therapy
Gene editing of hematopoietic stem cells
Hematopoietic stem cells: sourcing, selecting, culturing, and transducing
Learning from adverse events in gene therapy
Summary
Clinics care points
A Curative DNA Code for Hematopoietic Defects: Novel Cell Therapies for Monogenic Diseases of the Blood and Immune System
Key points
Introduction
Molecular interventions for correcting a gene
Genome editing mediated by DNA double-stranded breaks
Double-strand breaks repair pathways and the effect of cell cycle and chromatin structure on genome editing
Non-break mediated editing: base and primer editing
The science of genome editing the hematopoietic system
Persistence of the therapeutic stem cell product
Values and limitations in evaluating the therapeutic product in mouse models
Advancing this new class of medicine to the clinic
Clinics care points
Adeno-Associated Virus Vector Design–Moving the Adeno-Associated Virus to a Bioengineered Therapeutic Nanoparticle
Key points
Introduction
From virus to vector
Engineering vector genomes to improve efficacy, cargo capacity, and fate
Engineering the capsid to improve efficacy and selectivity
Summary and perspectives
Regulatory Aspects of Gene Therapy: The Regulatory Life Cycle
Key points
Introduction
Early phase interactions with the Food and Drug Administration
Early phase considerations for the development of gene therapy products in pediatric subjects
Available incentive programs
Summary
Chimeric Antigen Receptor T-cell Therapy: Current Status and Clinical Outcomes in Pediatric Hematologic Malignancies
Key points
Introduction
CD19-CART clinical trials and outcomes
Beyond CD19-targeting in B-cell malignancies
Toxicity profile
Limitations of current CART therapies and strategies to improve success
Beyond B-cell Acute Lymphoblastic Leukemia
Summary and Future Directions
Clinics care points
Funding support
Disclosure
Genome-Edited T Cell Therapies
Key points
Introduction
Addressing HLA Barriers for ‘Universal’ CAR T Cell Therapy Against B Cell Malignancies
Engineered T Cells for Non-B Lineage Hematological Malignancies
Addressing T cell exhaustion and persistence
Safety and long-term monitoring
Summary
Clinics care points
Funding & Disclosures
Gene-Based Natural Killer Cell Therapies for the Treatment of Pediatric Hematologic Malignancies
Key points
Background
Starting material for natural killer cell immunotherapy
Methods of Genetic Engineering in Natural Killer Cells
Therapeutic Applications of Genetically Modified Natural Killer Cells
Summary
Clinics care points
Gene Therapy for Hemoglobinopathies: Beta-Thalassemia, Sickle Cell Disease
Key points
Introduction
Summary
Hemophilia A/B
Key points
Introduction
Hemophilia gene therapy, the early years
Adeno-associated virus gene transfer
Lentiviral gene delivery
Gene editing for hemophilia
Discussion
Gene Therapy for Inborn Errors of Immunity: Severe Combined Immunodeficiencies
Key points
Introduction
X-severe combined immune deficiency
Adenosine deaminase-severe combined immune deficiency
Artemis-severe combined immune deficiency
RAG1-severe combined immune deficiency
IL-7 receptor (IL-7R) deficiency severe combined immune deficiency
JAK-3 severe combined immune deficiency
Clinics care points
Summary
Funding
Genes as Medicine: The Development of Gene Therapies for Inborn Errors of Immunity
Key points
Viral vector gene therapy
Summary
Clinics care points
Gene Therapy for Pediatric Neurologic Disease
Key points
Introduction
Ex vivo delivery
Complications and concerns
Future directions
Summary
Clinics care points
Disclosure
Ex Vivo and In Vivo Gene Therapy for Mucopolysaccharidoses: State of the Art
Key points
Introduction
Mucopolysaccharidosis type I, Hurler variant
Mucopolysaccharidosis type II, Hunter syndrome
Mucopolysaccharidosis type III, Sanfilippo syndrome
Mucopolysaccharidosis type IV, Morquio A syndrome
Mucopolysaccharidosis type VI, Maroteaux-Lamy syndrome
Other mucopolysaccharidoses
Summary
Conflict of interests

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

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Nirali N. Shah

Sung-Yun Pai