Online resource; title from PDF title page (Ebsco, viewed Dec. 18, 2014).

Includes bibliographical references and index.

Translating Gene Therapy to the Clinic, edited by Dr. Jeffrey Laurence and Michael Franklin, follows the recent, much-lauded special issue of Translational Research in emphasizing clinical milestones and critical barriers to further progress in the clinic. This comprehensive text provides a background for understanding the techniques involved in human gene therapy trials, and expands upon the disease-specific situations in which these new approaches currently have the greatest therapeutic application or potential, and those areas most in need of future research. It emphasizes methods, tools, and experimental approaches used by leaders in the field of translational gene therapy. The book promotes cross-disciplinary communication between the sub-specialties of medicine, and remains unified in theme.

Front Cover; Translating Gene Therapy to the Clinic: Techniques and Approaches; Copyright; Contents; Preface; REFERENCES; About the Editors; Contributors; Chapter 1 -- Translating Genome Engineering to Survival; 1.1 ORIGINS; 1.2 SYNCHRONICITY OF DISCOVERIES; 1.3 GENE ADDITION; 1.4 FROM GENE ADDITION TO GENE EDITING; 1.5 THERAPY FOR GENETIC DISORDERS; 1.6 ROADMAP TO THE FUTURE; CONFLICT OF INTEREST; ACKNOWLEDGMENTS; REFERENCES; Chapter 2 -- Pluripotent Stem Cells and Gene Therapy; 2.1 GENETIC APPROACHES TO PLURIPOTENCY; 2.2 TRANSCRIPTION FACTORS IMPORTANT FOR REPROGRAMMING TO PLURIPOTENCY.

2.3 METHODS FOR GENETIC REPROGRAMMING2.4 CLINICAL TRANSLATION OF IPSCS; 2.5 CONCLUSION; REFERENCES; Chapter 3 -- Genome Engineering for Therapeutic Applications; 3.1 INTRODUCTION; 3.2 CUSTOMIZABLE DNA-TARGETING PROTEINS; 3.3 GENOME EDITING WITH ENGINEERED NUCLEASES; 3.4 SYNTHETIC TRANSCRIPTION FACTORS FOR THERAPEUTIC APPLICATIONS; 3.5 CONCLUSION; ACKNOWLEDGMENTS; REFERENCES; Chapter 4 -- Immune System Obstacles to In vivo Gene Transfer with Adeno-Associated Virus Vectors; 4.1 INTRODUCTION; 4.2 AAV VECTORS; 4.3 INNATE IMMUNITY IN AAV GENE TRANSFER; 4.4 T-CELL RESPONSES TO VECTORS.

4.5 HUMORAL IMMUNITY4.6 CONCLUSIONS; ACKNOWLEDGMENT; REFERENCES; Chapter 5 -- Risks of Insertional Mutagenesis by DNA Transposons in Cancer Gene Therapy; 5.1 INSERTIONAL MUTAGENESIS-THE DOWNSIDE OF GENE THERAPY?; 5.2 SLEEPING BEAUTY TRANSPOSON/TRANSPOSASE SYSTEM ADAPTED FOR GENE THERAPY; 5.3 PLASTICITY OF GENOMES AND GENE EXPRESSION IN HUMANS; 5.4 TRANSPOSON-MEDIATED GENE THERAPY IN THE CLINIC; 5.5 CONCLUSIONS; ACKNOWLEDGMENT; REFERENCES; Chapter 6 -- Arthritis Gene Therapy: A Brief History and Perspective; 6.1 INTRODUCTION; 6.2 CONCEPTION AND STRATEGIES; 6.3 TECHNOLOGY DEVELOPMENT.

6.4 UNRESOLVED ISSUES6.5 CLINICAL TRIALS; 6.6 VETERINARY APPLICATIONS; 6.7 OTHER APPLICATIONS OF INTRA-ARTICULAR GENE THERAPY; 6.8 COMMERCIALIZATION; 6.9 PERSPECTIVES; ACKNOWLEDGMENTS; REFERENCES; Chapter 7 -- Type 1 Diabetes Mellitus: Immune Modulation as a Prerequisite for Successful Gene Therapy Strategies; 7.1 INTRODUCTION; 7.2 EFFECTIVE IMMUNE THERAPY/MODULATION: A PREREQUISITE FOR SUCCESSFUL GENE THERAPY OF TYPE 1 DIABETES; 7.3 TARGETED ISLET ANTIGEN RECOGNITION AND ANTIGEN-BASED THERAPIES; 7.4 BROAD IMMUNOSUPPRESSIVE THERAPIES; 7.5 IMMUNOTHERAPIES THAT TARGET EVENTS IN T CELL RESPONSE.