Translating Gene Therapy to the Clinic

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 field* Offers disease-based coverage from expert clinicians, including everything from arthritis to congestive heart failure , detailing specific progress and barriers for current translational use* Provides key background information from immune response through genome engineering and gene transfer, relevant information for practicing clinicians contemplating enrolling patients in gene therapy trials