Translating Gene Therapy to the Clinic: Techniques and Experimental Approaches

Translating Gene Therapy into the Clinic follows the recent, much-lauded special issue of Translational Research in emphasizing clinical milestones and critical barriers - particularly regulatory barriers - to further progress in the clinic. It significantly expands on that issue, by expanding upon the 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.  Pedagogically, the work provides an up-to-date resource on the latest knowledge, laboratory techniques, and experimental approaches used by translational research leaders in the field of gene therapy.  The book promotes cross-disciplinary communication between the sub-specialties of medicine, and remain unified in theme by emphasizing recent innovations, critical barriers to progress and the new tools that are being used to overcome them, and specific areas of research that require additional study to advance the field as a whole. Presents impactful and widely supported research across the spectrum of science, method, implementation and clinical application - reworked with new pedagogy and to review the broader fieldOffers disease-based coverage from expert clinicians, including everything from arthritis to congestive heart failure , detailing specific progress and barriers for current translational useProvides key background information from immune response through genome engineering and gene transfer, relevant information for practicing clinicians contemplating enrolling patients in gene therapy trials INDICE: 1. Introduction 2. Translating genome engineering to better clinical outcomes 3. Arthritis Gene Therapy and its Tortuous Path into the Clinic4. Mechanistic Basis of Immunotherapies for Type 1 Diabetes Mellitus 5. Gene therapy for retinal disease 6. Gene Therapy in Cystic Fibrosis 7. Evaluating Risks of Insertional Mutagenesis by DNA Transposons in Gene Therapy 8. Pluripotent Stem Cells and Gene Therapy 9. Gene therapy for hemoglobinopathies: progress and challenge 10. Hemophilia Clinical Gene Therapy-brief review 11. Gene Transfer for Congestive Heart Failure12. Gene therapy for the prevention of vein graft disease 13. Current status of gene therapy for brain tumors 14. Oncolytic virus therapy for cancer: The first wave of translational clinical trials 15. T-Cell Gene Therapy 16. Immune responses to gene therapy

• ISBN: 978-0-12-800563-7
• Editorial: Academic Press
• Encuadernacion: Cartoné
• Fecha Publicación: 15/03/2015
• Nº Volúmenes: 1
• Idioma: Inglés