Translating Gene Therapy to the Clinic
Techniques and Approaches

Reviews the latest research in gene transfer, genome engineering and clinical trials, as gene-based therapeutic techniques move from the lab to the clinic

Jeffrey Laurence (Edited by), Michael Franklin (Edited by)

9780128005637

Hardback, published 22 December 2014

346 pages, 60 illustrations
27.6 x 21.5 x 2.5 cm, 1.16 kg

"This is a unique book, not only because of the information it provides, but also because of the way it presents it. The editors have done a wonderful job of recruiting an excellent group of contributors and designing a wonderful book. Score: 100 - 5 Stars" --Doody's

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 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

Subject Areas: Molecular biology [PSD], DNA & Genome [PSAK1], Oncology [MJCL], Medical genetics [MFN]