In this monograph about gene therapy of autoimmune and inflammatory disorders
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 hurdle
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 natural
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 clinicians
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.