Concepts in Gene Therapy (PDF)

Frontmatter -- Preface -- Contents -- I. Methods of Gene Delivery -- 1. Retroviral Vectors -- 2. Adenoviral Vectors -- 3. Herpes Simplex Virus Mediated Gene Transfer to Neurons -- 4. Adeno-Associated Viral Vectors -- 5. Virus Functions for Artificial Vectors -- 6. Targeted Gene Delivery and Expression in Hepatocytes -- 7. Particle-Mediated Gene Delivery System for Cancer Research -- II. Methods for Inactivation or Restoration of Gene Function -- 8. Gene Inactivation by Homologous Recombination - Animal Models of Plasma Lipoprotein Disorders -- 9. Potential Antisense Oligonucleotide Therapies for Neurodegenerative Diseases -- 10. Synthetic Ribozymes: The Hammerhead Ribozyme -- 11. Ribozymes as Tools for the Gene Therapist -- III. Cellular Target Systems -- 12. Gene Transfer and Transgene Expression in Hematopoietic Cells -- 13. The Liver as a Target for Gene Therapy -- 14. Gene Therapy for Gaucher Disease via Genetically Engineered Primary Myoblasts -- 15. In vivo Secretion of Therapeutic Proteins from Neo-Organs -- IV. Genetic Diseases -- 16. Gene Therapy for Cystic Fibrosis - Strategies, Problems and Perspectives -- 17. Gene Therapy for Dystrophin Deficiency -- 18. Gene Therapy For Familial Hypercholesterolemia -- 19. Therapeutic Strategies for the Lysosomal Storage Disorders -- V. Non-Genetic and Infectious Diseases -- 20. Gene Therapy for Arthritis -- 21. Gene Therapy for HIV-1 Disease -- VI. Cancer -- 22. Suicide Genes for the Treatment of Cancer -- 23. Tumor Cell Vaccines Using Genetically Modified Cells Coexpressing Cytokines and the T Cell Costimulatory Molecule B7 -- 24. Dosage Impact on Immunotherapy with Cytokine-Gene Modified Cancer Vaccines -- 25. Tumor Suppressor Gene Therapy - Growth Arrest and Programmed Cell Death -- Acknowledgement -- References -- Contributors -- Index