Title Somatic gene therapy / edited by Patricia L. Chang

Published Boca Raton, FL : CRC Press, Taylor & Francis Group, [2018]

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Description 1 online resource (314 pages) Series CRC revivals CRC revivals Contents 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 Summary "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 Bibliography Includes bibliographical references and index Notes III. Epidermal-Specific Disease: Treatment Via Homologous Recombination Print version record Subject Gene therapy. Genetic Therapy HEALTH & FITNESS -- Diseases -- General. MEDICAL -- Clinical Medicine. MEDICAL -- Diseases. MEDICAL -- Evidence-Based Medicine. MEDICAL -- Internal Medicine. Gene therapy Form Electronic book Author Chang, Patricia L., editor ISBN 9781351093668 1351093665 9781351076760 1351076760 9781351085212 1351085212 9781351102117 1351102117

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