Somatic gene therapy

Description

"As human gene therapy becomes a clinical reality, a new era in medicine dawns. Novel and innovative developments in molecular genetics now provide opportunities to treat the genetic bases of diseases often untreatable before. Somatic Gene Therapy documents these historical clinical trials, reviews current advances in the field, evaluates the use of the many different cell types and organs amenable to gene transfer, and examines the prospects of various exciting strategies for gene therapy."--Provided by publisher. Read more... Abstract: "As human gene therapy becomes a clinical reality, a new era in medicine dawns. Novel and innovative developments in molecular genetics now provide opportunities to treat the genetic bases of diseases often untreatable before. Somatic Gene Therapy documents these historical clinical trials, reviews current advances in the field, evaluates the use of the many different cell types and organs amenable to gene transfer, and examines the prospects of various exciting strategies for gene therapy."--Provided by publisher Content: 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 TherapyA. 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 SCIDC. CD3-γ 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. III. Genetic Engineering of Human FibroblastsIV. Disease Models: Implantation of Genetically-Modified Fibroblasts V. Implantation of Fibroblasts in Tandem with Substrates VI. Conclusion: Role of Fibroblasts in Gene Therapy References Chapter 5: Hepatic Gene Therapy I. Introduction II. Methods for Gene Delivery to the Liver A. Ex Vivo Gene Delivery to Hepatocytes B. In Vivo Transduction with Retroviral Vectors C. In Vivo Gene Transfer Using Recombinant Adenoviral Vectors D. Hepatic Delivery of DNA-Based Vectors E. Future Directions for Gene Delivery to the Liver. III. Approaching Clinical Applications of Hepatic Gene TherapyIV. Conclusion Acknowledgments References Chapter 6: Epidermal Keratinocytes: Opportunities and Applications in Somatic Cell Gene Therapy I. Epidermal Keratinocytes: Target Cells for Gene Therapy A. In Vivo Characteristics B. Keratinocyte Culture Systems II. Treatment of Nonepidermal Genetic Disease with Keratinocytes A. Experimental Systems Suggest In Vivo Success B. Selection and Delivery of Therapeutic Genes C. Promoter Choices for Epidermal Keratinocyte-Based Gene Therapy.