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

Nirali N. Shah, Sung-Yun Pai (Herausgeber)

Buch | Hardcover

240 Seiten

2022
Elsevier - Health Sciences Division (Verlag)
978-0-323-98775-2 (ISBN)

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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.

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.

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

Erscheinungsdatum	17.08.2022
Reihe/Serie	The Clinics: Internal Medicine
Verlagsort	Philadelphia
Sprache	englisch
Maße	152 x 229 mm
Gewicht	540 g
Themenwelt	Medizinische Fachgebiete ► Innere Medizin ► Hämatologie
	Medizin / Pharmazie ► Medizinische Fachgebiete ► Onkologie
	Medizin / Pharmazie ► Medizinische Fachgebiete ► Pädiatrie
	Medizin / Pharmazie ► Studium
ISBN-10	0-323-98775-3 / 0323987753
ISBN-13	978-0-323-98775-2 / 9780323987752
Zustand	Neuware