Few areas of biomedical research provide greater opportunities for radically new therapies for devastating diseases that have evaded treatment so far than gene therapy. This is particularly true for the brain and nervous system, where gene transfer has become a key technology for basic research and has recently been translated to human therapy in several landmark clinical trials. Gene Therapy of the Central Nervous System: From Bench to Bedside represents the first definitive volume on this subject. Edited by two pioneers of neurological gene therapy, this volume contains contributions by leaders who helped create this field and are expanding the promise of gene therapy for the future of basic and clinical neuroscience. Drawing upon this extensive collective experience, this book provides clear and informative reviews on a variety of subjects of interest to anyone exploring or using gene therapy for neurobiological applications in research and clinical praxis.

Key Features

* Presents gene transfer technologies with particular emphases upon novel vehicles, immunological issues and the role of gene therapy in stem cells * Discusses preclinical areas that are likely to translate into clinical studies in the near future, including epilepsy, pain and amyotrophic lateral sclerosis * Includes "insider" information on technological and regulatory issues which can often limit effective translation of even the most promising idea into clinical use


Neuroscientists, geneticists, molecular biologists, pharmaceutical companies

Table of Contents

SECTION I. GENE TRANSFER TECHNOLOGY AND REGULATORY ISSUES 1. Design and optimization of expression cassettes including promoter choice and regulatory lements 2. Identification of Novel AAV Serotypes for Use as Vectors 3. HSV amplicon vectors for gene delivery to the nervous system 4. Influence of the immune system on CNS gene transfer 5. Targeted induction of endogenous neural stem and progenitor cells: A new strategy for gene therapy of neurological disease 6. Neurosurgical Targeting, Delivery and Infusion of Gene Therapy Agents in the Brain 7. Gene Transfer for Neurological Disease: Agencies, Policies and Process SECTION II. GENE THERAPY FOR DEGENERATIVE AND FUNCTIONAL DISORDERS 8. Gene therapy for Parkinson’s disease 9. Non human primate models for testing gene therapy for neurodegenerative disorders 10. Delivery of Molecular Therapeutics into the CNS and their Distribution within the Brain 11. Gene therapy for CNS diseases using intrabodies 12. Gene therapy for epilepsy SECTION IIII. PSYCHIATRIC AND BEHAVIORAL GENE THERAPY 13. Genetic manipulation of learning and memory 14. Psychiatric applications of viral vectors 15. Use of viral vectors to influencebehavior SECTION IV. GENE THERAPY FOR PAIN AND SPINAL CORD DISEASES 16. Herpes simplex vector mediated gene transfer to the peripheral nervous system for the treatment of polyneuropathy and chronic pain 17. Characterization of pain receptors in the spinal cord using a viral vector for spatial-temportal gene targeting 18. Viral vector mediated gene therapy of pain 19. VEGF, an angiogenic factor with neurotrophic activity, useful for treatment of ALS? 20. Viral Vec


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© 2006
Academic Press
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About the editors

Michael Kaplitt

Affiliations and Expertise

New York Hospital, Cornell University Medical College and Laboratory of Neurobiology and Behavior, The Rockefeller University, New York, U.S.A.