Cover -- Half Title -- Title Page -- Copyright Page -- PREFACE -- THE EDITOR -- CONTRIBUTORS -- Table of Contents -- Chapter 1: Overview -- I. The Somatic Tissue -- II. The Delivery System -- A. Physico-Chemical Methods -- B. Biological Vectors -- III. The Diseases -- References -- Chapter 2: Human Hematopoietic Cells for Gene Therapy -- I. Introduction -- II. Isolation, Characterization, and Ex Vivo Maintenance of Human Primitive Hematopoietic Cells -- A. Techniques for the Enrichment of Human Primitive Hematopoietic Cells -- B. Ex Vivo Maintenance and Expansion of Human Primitive Hematopoietic Cells -- III. Development of Preclinical Models for Gene Therapy -- A. Murine Models for Gene Therapy of Hematologic Diseases -- B. Large Animal Models for Gene Therapy of Hematologic Diseases -- C. Gene Transfer into Human Hematopoietic Cells -- IV. Human Gene-Marking Clinical Trials -- V. Human Gene Therapy Clinical Trials -- VI. Conclusion -- References -- Chapter 3: Somatic Gene Therapy for Severe Combined Immune Deficiency (SCID) -- I. Biology and Clinical Manifestations of SCID -- A. Demographic Features -- B. Clinical Features -- C. Laboratory Findings -- II. Pathogenesis of SCID -- A. Adenosine Dearninase (ADA) Deficiency -- B. X-Linked SCID -- C. CD3-Î3 Deficiency -- D. ZAP-70 Deficiency -- E. Signaling Defects and IL-2 Deficiency Syndromes -- F. Major Histocompatibility Complex Defects -- III. Treatments for SCID -- A. Bone Marrow Transplantation (BMT) -- B. Enzyme Replacement Therapy -- IV. Gene Therapy for SCID -- A. Theoretical Considerations -- B. Preclinical Studies -- C. Clinical Studies of Gene Therapy for ADA-Deficient SCID -- V. Considerations for Gene Therapy of Other Forms of SCID -- Acknowledgments -- References -- Chapter 4: Fibroblast Cell Biology and Gene Therapy -- I. Introduction -- II. Growth of Fibroblasts In Vitro