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Translating gene therapy to the clinic : techniques and approaches /
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 backgrou...
| Clasificación: | Libro Electrónico |
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| Otros Autores: | , |
| Formato: | Electrónico eBook |
| Idioma: | Inglés |
| Publicado: |
Amsterdam :
Academic Press, an imprint of Elsevier,
[2015]
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| Colección: | Advances in translational research.
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| Temas: | |
| Acceso en línea: | Texto completo |
Tabla de Contenidos:
- 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.