مفاهیم در ژن درمانی ۲۰۲۰
Concepts in Gene Therapy 2020

1. Preface

2. Contents

3. 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

11. 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

16. 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

21. 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

26. V. Non-Genetic and Infectious Diseases

20. Gene Therapy for Arthritis

21. Gene Therapy for HIV-1 Disease

29. 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

34. Acknowledgement

35. References

36. Contributors

37. Index