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.
From the reviews: "This monograph contains new and valuable information regarding relevant topics ranging from disease entities to vectors and engineered cells. The authors, all of them international experts, take into consideration the biomedical knowledge of these diseases and address the complexities of delivering long term genetic interventions. ... The present volume is of an exceptional value for clinicians and scientists interested in introducing gene therapy into clinical use." (Magdalena Moisei, Romanian Journal of Biochemistry, Vol. 47 (2), 2010)
"The information is quite extensive and useful. Each chapter is well referenced. This monograph is highly specialized but is easily understandable. It is recommended for all researchers - both clinical and non-clinical, particularly those working in the fields of gene therapy, immunology, rheumatology, pharmacology and molecular medicine. It will also be a useful reference source for the libraries of all advanced academic centers and research institutions." (S. S. Agarwal, Indian Journal of Medical Research, Vol. 135, April, 2012)
"This monograph contains new and valuable information regarding relevant topics ranging from disease entities to vectors and engineered cells. The authors, all of them international experts, take into consideration the biomedical knowledge of these diseases and address the complexities of delivering long termgenetic interventions. ... The present volume is of an exceptional value for clinicians and scientists interested in introducing gene therapy into clinical use." (Magdalena Moisei, Romanian Journal of Biochemistry, Vol. 47 (2), 2010)
"This monograph contains new and valuable information regarding relevant topics ranging from disease entities to vectors and engineered cells. The authors, all of them international experts, take into consideration the biomedical knowledge of these diseases and address the complexities of delivering long termgenetic interventions. ... The present volume is of an exceptional value for clinicians and scientists interested in introducing gene therapy into clinical use." (Magdalena Moisei, Romanian Journal of Biochemistry, Vol. 47 (2), 2010)