Blood Cell Biochemistry

1. Front Matter

   Pages i-xvi

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2. Hemopoietic Stem Cells as Targets for Genetic Manipulation

   • N. G. Testa, E. A. de Wynter, J. Hows

   Pages 1-12

3. Bone Marrow Transplantation for Genetic Diseases

   • Colin G. Steward

   Pages 13-56

4. Retroviral Vectors

   • Mary Collins, Colin Porter

   Pages 57-88

5. Parvoviral Vectors for Human Hematopoietic Gene Therapy

   • Arun Srivastava

   Pages 89-122

6. Nonviral Methods for Gene Transfer

   • A. Djeha, L. S. Lashford

   Pages 123-154

7. Prospects for Gene Therapy of Inherited Immunodeficiency

   • Colin Casimir

   Pages 155-202

8. Gene Therapy for Lysosomal Disorders

   • L. J. Fairbairn, L. S. Lashford

   Pages 203-218

9. Genetic Approaches to Therapy for the Hemoglobinopathies

   • Michael Antoniou, Frank Grosveld

   Pages 219-242

10. Gene Marking and the Biology of Hematopoietic Cell Transfer in Human Clinical Trials

    • A. K. Stewart, I. D. Dubé, R. G. Hawley

    Pages 243-268

11. Antisense Strategies to Leukemia

    • Stephen G. O’Brien

    Pages 269-292

12. Transfer of Drug Resistance Genes into Bone Marrow Stem and Progenitor Cells: Implications for Cancer Chemotherapy

    • J. A. Rafferty, L. J. Fairbairn

    Pages 293-312

13. HIV Gene Therapy Using Hairpin Ribozymes in Hematopoietic Stem/Progenitor Cells

    • Xinqiang Li, Flossie Wong-Staal, Anthony D. Ho, Ping Law

    Pages 313-330

14. Molecular Immunotherapy by Gene Transfer

    • Rosa Maria Diaz, Richard G. Vile

    Pages 331-350

15. DNA-Based Immunization

    • Heather L. Davis, Cynthia L. Brazolot Millan

    Pages 351-376

16. Back Matter

    Pages 377-380

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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.

• HIV
• biochemistry
• gene therapy
• genes
• tissue
• transplantation

• Christie CRC Research Centre Paterson Institute for Cancer Research, Christie Hospital NHS Trust, Manchester, UK

  Leslie J. Fairbairn, Nydia G. Testa

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