This book deals with the various aspects of gene therapy from the point of view of leading experts in their respective fields. It provides not only a perfect overview of the various topics of gene therapy but also discusses current problems and potential solutions. The various gene delivery vehicles and model systems for diseases are described in detail and specific problems encountered in the individual target diseases (genetic and nongenetic diseases, as well as AIDS and cancer) are discussed in depth. The book is essential reading for students, scientists and physicians interested in molecular medicine.…mehr
This book deals with the various aspects of gene therapy from the point of view of leading experts in their respective fields. It provides not only a perfect overview of the various topics of gene therapy but also discusses current problems and potential solutions. The various gene delivery vehicles and model systems for diseases are described in detail and specific problems encountered in the individual target diseases (genetic and nongenetic diseases, as well as AIDS and cancer) are discussed in depth. The book is essential reading for students, scientists and physicians interested in molecular medicine.
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
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
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