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Blood Cell Biochemistry : Hematopoiesis and Gene Therapy - Leslie J. Fairbairn

Blood Cell Biochemistry

Hematopoiesis and Gene Therapy

By: Leslie J. Fairbairn (Editor), Nydia G. Testa (Editor)

Hardcover

Published: 31st March 1999
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Since the first concepts of gene therapy were formulated, the hemopoietic system has been considered the most natural first target tissue for genetic manipulation. The reasons for this include the fact that a very large number of inherited disorders (including some of the most common disorders, such as the hemoglobinopathies) are disorders of the hemopoietic system, and the large amount of experience in hematopoietic transplantation biology. The consequence of this resulted in the first clinical trial of gene therapy in 1989, where two children suffering from severe combined immune deficiency (ADA-SCID) were transplanted with T-cells express­ ing adenosine deaminase (the defective enzyme in patients with this disorder). The partial success of this treatment was perhaps responsible for undue optimism among those proposing other gene therapy treatments within the hematopoietic system, and it has since become clear that there are a number of technical and biological difficulties to overcome before hematopoietic gene therapy becomes a mainstream therapeutic strategy. The chapters in this book evaluate the need for gene therapy in the hematopoietic system, discuss how efficient gene transfer and expression can be achieved in the target cells, highlight areas of difficulty to be addressed, and examine a number of potential applications of the gene therapy approach. The book begins with a chapter by Testa and colleagues, discussing the various sources of hematopoietic cells for both transplantation and gene therapy.

Hematopoietic Stem Cells as Targets for Genetic Manipulation: Concepts and Practical Approaches
Bone Marrow Transplantation for Genetic Diseases
Retroviral Vectors
Parvoviral Vectors for Human Hematopoietic Gene Therapy
Nonviral Methods for Gene Transfer
Prospects for Gene Therapy of Inherited Immunodeficiency
Gene Therapy for Lysosomal Disorders
Genetic Approaches to Therapy for the Hemoglobinopathies
Gene Marking and the Biology of Hematopoietic Cell Transfer in Human Clinical Trials
Antisense Strategies to Leukemia
Transfer of Drug Resistance Genes into Bone Marrow Stem and Progenitor Cells: Implications for Cancer Chemotherapy
HIV Gene Therapy Using Hairpin Ribozymes in Hematopoietic Stem Progenitor Cells
Molecular Immunotherapy by Gene Transfer
DNA-Based Immunization
Table of Contents provided by Publisher. All Rights Reserved.

ISBN: 9780306459627
ISBN-10: 0306459620
Series: BLOOD CELL BIOCHEMISTRY : Book 8
Audience: Professional
Format: Hardcover
Language: English
Number Of Pages: 380
Published: 31st March 1999
Publisher: Springer Science+Business Media
Country of Publication: US
Dimensions (cm): 17.15 x 27.31  x 3.18
Weight (kg): 0.98