Gene Therapy of the Central Nervous System: From Bench to Bedside

Edited by

  • Michael Kaplitt, New York Hospital, Cornell University Medical College and Laboratory of Neurobiology and Behavior, The Rockefeller University, New York, U.S.A.
  • Matthew During

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.
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Neuroscientists, geneticists, molecular biologists, pharmaceutical companies


Book information

  • Published: November 2005
  • ISBN: 978-0-12-397632-1

Table of Contents

SECTION I. GENE TRANSFER TECHNOLOGY AND REGULATORY ISSUES1. Design and optimization of expression cassettes including promoter choice and regulatory lements 2. Identification of Novel AAV Serotypes for Use as Vectors3. HSV amplicon vectors for gene delivery to the nervous system4. Influence of the immune system on CNS gene transfer5. Targeted induction of endogenous neural stem and progenitor cells: A new strategy for gene therapy of neurological disease6. Neurosurgical Targeting, Delivery and Infusion of Gene Therapy Agents in the Brain7. Gene Transfer for Neurological Disease: Agencies, Policies and ProcessSECTION II. GENE THERAPY FOR DEGENERATIVE AND FUNCTIONAL DISORDERS8. Gene therapy for Parkinson’s disease9. Non human primate models for testing gene therapy for neurodegenerative disorders10. Delivery of Molecular Therapeutics into the CNS and their Distribution within the Brain11. Gene therapy for CNS diseases using intrabodies12. Gene therapy for epilepsy SECTION IIII. PSYCHIATRIC AND BEHAVIORAL GENE THERAPY13. Genetic manipulation of learning and memory14. Psychiatric applications of viral vectors15. Use of viral vectors to influencebehaviorSECTION IV. GENE THERAPY FOR PAIN AND SPINAL CORD DISEASES16. Herpes simplex vector mediated gene transfer to the peripheral nervous system for the treatment of polyneuropathy and chronic pain17. Characterization of pain receptors in the spinal cord using a viral vector for spatial-temportal gene targeting18. Viral vector mediated gene therapy of pain19. VEGF, an angiogenic factor with neurotrophic activity, useful for treatment of ALS?20. Viral Vector Axonal Uptake and Retrograde Transport: Mechanisms and Applications21. Gene therapy for spinal cord injury V. GENE THERAPY FOR BRAIN TUMORS AND NEUROGENETIC DISEASES22. Prodrug-Activation Gene Therapy 23. Clinical trials of gene therapy for Canavan Disease 24. Gene therapy for the late infantile form of Batten disease 25. Molecular Imaging of Gene Therapy for Neurogenetic Diseases