Gene Therapy for Autoimmune and Inflammatory Diseases

In this monograph about gene therapy of autoimmune and inflammatory d- orders we have gathered international experts and leaders from different fields to review the state of the art advances on topics ranging from disease entities to vectors and engineered cells. The different approaches described in each chapter take into consideration the biomedical knowledge of these diseases and address the complexities of delivering long-term genetic interventions. Gene therapy also serves as a testing ground for new therapeutic entities and helps provide proof of principle for their potential therapeutic role in animal models of disease. Scaling up from mice to men still remains an important h- dle not only from the quantitative point of view, but also for currently unknown and unexpected secondary effects of the vector or the transgene. Some of these approaches have already been tested in the clinic, but much more needs to be done to understand the human conditions treated and the n- ural history of their pathology. We are indebted to the secretarial assistance of Ms. Lin Wells (Bone and Joint Research Unit, London, UK) and the help of Hans Detlef Klüber for his help in getting this book published. We hope this book will be of interest to c- nicians and scientists and inspiring to students of the subject who will use their own ingenuity and knowledge to further forward this discipline into clinical use.

This text addresses the latest advances in viral and non-viral vectors as well as novel targets and methods for the treatment of autoimmune and inflammatory disease. Its main focus is on pre-clinical testing with up-to-date clinical trials.

Gene therapy for arthritis.- Gene therapy for the treatment of inflammatory bowel disease.- Gene therapy for diabetes.- Gene therapy for cystic fibrosis lung disease.- Gene therapy of multiple sclerosis.- Gene therapy for myositis.- Gene therapy for osteoarthritis.- Gene therapy: Sjögren's syndrome.- Disease mechanisms, genetic susceptibility and therapeutic approaches in lupus disease.- Regulated promoters.- Development of AAV vectors for the therapy of autoimmune and inflammatory diseases.- Delivery and application of plasmid DNA in arthritis gene therapy.- Helper-dependent adenoviral vectors.- Cells as carriers of gene therapy.- Perspectives for the future developments of gene therapy for autoimmune and inflammatory therapy.