Gene Therapy for Autoimmune and Inflammatory Diseases

1. Front Matter

   Pages I-XI

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2. Gene therapy for arthritis

   • Florence Apparailly, Paul Peter Tak, Christian Jorgensen

   Pages 1-18

3. Gene therapy for the treatment of inflammatory bowel disease

   • Melanie A. Ruffner, Scott E. Plevy, Anthony T. Cheung

   Pages 19-37

4. Gene therapy for diabetes

   • Hirofumi Noguchi

   Pages 39-46

5. Gene therapy for cystic fibrosis lung disease

   • Stephanie G. Sumner-Jones, Deborah R. Gill, Stephen C. Hyde

   Pages 47-64

6. Gene therapy of multiple sclerosis

   • Roberto Furlan, Chiara Maiorino, Alberto Gatta, Francesca Ruffini, Gianvito Martino

   Pages 65-78

7. Gene therapy for myositis

   • Hans H. Jung, Juliane Bremer, Michael Weller

   Pages 79-90

8. Gene therapy for osteoarthritis

   • Steven C. Ghivizzani, Christopher H. Evans

   Pages 91-112

9. Gene therapy: Sjögren’s syndrome

   • Nienke Roescher, Paul Peter Tak, John A. Chiorini

   Pages 113-125

10. Disease mechanisms, genetic susceptibility and therapeutic approaches in lupus disease

    • Rizgar A. Mageed, Taher E. Taher, Ali S. Jawad

    Pages 127-146

11. Regulated promoters

    • Jeroen Geurts, Wim B. van den Berg, Fons A. J. van de Loo

    Pages 147-159

12. Development of AAV vectors for the therapy of autoimmune and inflammatory diseases

    • Chengwen Li, Matthew L. Hirsch, R. Jude Samulski

    Pages 161-180

13. Delivery and application of plasmid DNA in arthritis gene therapy

    • Maria C. Subang, David J. Gould

    Pages 181-192

14. Helper-dependent adenoviral vectors

    • Nicola Brunetti-Pierri, Philip Ng

    Pages 193-207

15. Cells as carriers of gene therapy

    • Ingo H. Tarner

    Pages 209-222

16. Perspectives for the future developments of gene therapy for autoimmune and inflammatory therapy

    • Yuti Chernajovsky, Paul D. Robbins

    Pages 223-231

17. Back Matter

    Pages 233-244

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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.

• Arthritis
• Autoimmune Diseases
• DNA
• Immunology
• Inflammatory Diseases
• Promoter
• gene therapy
• genes

“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)

• Bone and Joint Research Unit Barts and The London School of Medicine, Queen Mary University of London, London, UK

  Yuti Chernajovsky

• Department of Microbiology and Molecular Genetics, University of Pittsburgh School of Medicine, Pittsburgh, USA

  Paul D. Robbins

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