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 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.

Presents impactful and widely supported research across the spectrum of science, method, implementation and clinical application Offers disease-based coverage from expert clinician-scientists, covering everything from arthritis to congestive heart failure, as it details 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

1. Introduction
2. Translating genome engineering to better clinical outcomes
3. Arthritis Gene Therapy and Its Tortuous Path into the Clinic
4. 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 Failure
12. 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