Buchumschlag

Gene therapy of the central nervous system: from bench to bedside

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. Inf...

Ausführliche Beschreibung

Gespeichert in:
Bibliographische Detailangaben
Weitere Verfasser: Kaplitt, Michael G. (HerausgeberIn)
Dokumenttyp: Book/Monograph
Sprache:Englisch
Veröffentlicht: Amsterdam Boston Academic Press 2006
Volumes / Articles: Show Volumes / Articles.
Schlagworte:
Electronic books
Online-Zugang:Verlag, lizenzpflichtig: https://www.sciencedirect.com/science/book/9780123976321
Verlag, ScienceDirect, Volltext: http://www.sciencedirect.com/science/book/9780123976321
Volltext
Verfasserangaben:edited by Michael G. Kaplitt and Matthew J. During
Inhaltsangabe:
  • Cover; Gene Therapy of the Central Nervous System: From Bench to Bedside; Contents; Contributors; Preface; Section I Gene Transfer Technology and Regulatory Issues; Design and Optimization of Expression Cassettes Including Promoter Choice and Regulatory Elements; Introduction; Design of the rAAV Cassette; Cell-Type-Specific Tropism of rAAV; Choice of Promoter; Regulatory Elements; Summary and Conclusion; Identification of Novel Adeno-Associated Virus Serotypes for Use as Vectors; Introduction; Identification and Isolation of Novel Primate AAVs; Classification of Novel Primate AAVs
  • Novel AAVs as Gene Transfer VectorsConclusions; HSV Amplicon Vectors for Gene Delivery to the Nervous System; Basics of HSV-1 Amplicon; Advantages of HSV-1 Amplicon; Uses in Nervous System; Perspectives; Acknowledgments; Influence of the Immune System on Central Nervous System Gene Transfer; Adaptive Immunity and Viral Vectors; Immunology of Transgenes; Conclusion; Acknowledgments; Targeted Induction of Endogenous Neural Stem and Progenitor Cells: A New Strategy for Gene Therapy of Neurological Disease; Neural Stem and Progenitor Cells of the Human Brain
  • Cell Genesis in the Adult CNS Shares Common Themes with that in other Solid OrgansNeural Progenitor Cells are widely Distributed in the Adult Human Brain; Progenitor Cells may be useful as both Engraftable and Inducible Substrates for Repair; Endogenous Progenitor Cells are Mobilized by Injury and Disease; Resident Progenitor Cells can be Mobilized Pharmacologically and Genetically; Induced Neurogenesis in the Adult Neostriatum; The Development of Glial Suppressive Strategies Enhancing Heterotopic Neurogenesis; Induced Neurogenesis as a Therapeutic Strategy in Huntington's Disease
  • Progenitor Cell Targeting in Parkinson's DiseaseInduced Neurogenesis as a Restorative Strategy for the Hippocampal Atrophies; Parenchymal Glial Progenitors are Attractive Targets for Exogenous Mobilization; Transduced Neural Progenitors as Vectors for Enzymatic Repletion in the Storage Diseases; Overview; Acknowledgments; Neurosurgical Targeting, Delivery, and Infusion of Gene Therapy Agents in the Brain; Anatomical Targeting in Stereotactic Neurosurgery; Methods of Accessing the Central Nervous System; Methods for Enhancing Intracranial Gene Transfer; Conclusion
  • Gene Transfer for Neurological Disease: Agencies, Policies, and Process*The Landscape of Gene Therapy; Oversight of Gene Therapy Protocols; Good Practice Requirements for Gene Therapy Protocols; Clinical Phases for Development of Biologics; Sequential Steps in Implementation of a Clinical Gene Transfer Trial; Limitations of the System; Section II Gene Therapy for Degenerative and Functional Disorders; Gene Therapy for Parkinson's Disease; Introduction; Parkinson's Disease; Current Pharmacological Therapy vs. Gene Therapy for PD; Animal Models of PD
  • Strategies for Gene Therapy Intervention in PD
  • 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 Parkinsons 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 peripheralnervous 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 Vector Axonal Uptake and Retrograde Transport: Mechanisms and Applications
  • 21. Gene therapy for spinal cord injury
  • V. GENE THERAPY FOR BRAIN TUMORS AND NEUROGENETIC DISEASES
  • 22. 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

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