Translating gene therapy to the clinic : techniques and approaches / / edited by Jeffrey Laurence, Michael Franklin

• Pubblicazione: London, England : , : Academic Press, , 2015; ©2015
• Descrizione fisica: 1 online resource (347 p.)
• Disciplina: 616.042
• Soggetto topico: Gene therapy; Human genetics
• Persona (resp. second.): LaurenceJeffrey; FranklinMichael
• Note generali: Description based upon print version of record.
• Nota di bibliografia: Includes bibliographical references at the end of each chapters and index.
• Nota di contenuto: 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 ; 7.6 PROSPECTS FOR IMMUNOTHERAPY IN PROTECTING NEO-BETA CELLS7.7 CONCLUSION; ACKNOWLEDGMENT; REFERENCES; Chapter 8 - Gene Therapy for Diabetes; LIST OF ABBREVIATIONS; 8.1 INTRODUCTION; 8.2 GENERATION OF Β CELLS FROM PANCREATIC MATURE NON-Β CELLS; 8.3 GENERATION OF Β CELLS FROM TISSUE PROGENITOR CELLS; 8.4 GENERATION OF Β CELLS FROM STEM CELLS; 8.5 GENERATION OF NEW Β CELLS BY INDUCING THEIR REPLICATION; 8.6 CLOSING REMARKS; ACKNOWLEDGMENTS; REFERENCES; Chapter 9 - Gene Therapy for Neurological Diseases; 9.1 INTRODUCTION; 9.2 VIRAL VECTORS FOR NEUROLOGICAL DISEASES ; 9.3 GENE THERAPY FOR CHRONIC PAIN
• Sommario/riassunto: 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,
• Titolo autorizzato: Translating gene therapy to the clinic
• ISBN: 0-12-800564-5
• Formato: Materiale a stampa
• Livello bibliografico: Monografia
• Lingua di pubblicazione: Inglese
• Record Nr.: 9910787927203321
• Lo trovi qui: Univ. Federico II