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.
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