GENE THERAPY - THE STATE OF THE ART

Contents :

• Foreword

• ABSTRACT

• 1. INTRODUCTION

• 2. BASIC GENETICS OF HUMAN DISEASES

• 2.1 Autosomal Dominant  Inheritance

• 2.2 Autosomal Recessive Inheritance

• 2.3 X-Linked Inheritance

• 2.4 Multifactorial Disorders

• 3. MOLECULAR BASIS OF HUMAN GENTICS DISEASES

• 3.1 Structure and Function of DNA

• 3.2 Gene Expression

• 3.3 Recombinant DNA technology

• 3.3.1 Making Recombinant DNA

• 3.3.2 Restrcition Enzymes

• 3.3.3 Vectors

• 3.3.4 Cloning DNA

• 4. TYPES OF GENE THERAPY

• 4.1 Somatic Gene Therapy

• 4.2 Germline gene therapy

• 5. TARGET CELLS

• 5.1 In Vivo Gene Therapy

• 5.2 Ex-Vivo (In-vitro) Gene Therapy

• 5.3 The Bone Marrow Cells

• 5.4 Skin Cells

• 5.5 Liver cells or hypatocytes

• 5.6 Retinal Pigment Epithelial Cells

• 5.7 T-Cells

• 5.8 Stem Cells

• 6. THE TECHNOLOGY OF GENE THERAPY

• 7. DELIVERY SYSTEMS TO THE APPROPRIATE TISSUE

• 7.1 Viral Vectors for Gene Therapy

• 7.1.1 Retrovirus Vectors

• 7.1.1.1 The retroviral genome

• 7.1.1.2 The use of retrovirus for gene therapy

• 7.1.2 Adenovirus Vectors

• 7.1.2.1 Viral Genome

• 7.1.2.2 Life Cycle

• 7.1.2.3 Construction of vector

• 7.1.2.4 Replication of Adenoviral vectors

• 7.1.2.5 Cell Targeting of Adenovirus

• 7.1.2.6 Advantages and Disadvantages of Adenovirus

• 7.1.3 Adeno Associated viruses AAV

• 7.1.3.1 Construction of AAV

• 7.1.3.2 Herpes Simplex Virus HSV

• 7.2 Non Viral Systems for Gene Therapy

• 7.2.1 Non-Viral Vectors Used in In-Vivo Applications

• 7.2.1.1 Calcium Phosphate

• 7.2.1.2 Microinjection

• 7.2.1.3 Electroporation

• 7.2.2 Non-Viral Techniques with both In Vitro and In Vivo Applications

• 7.2.2.1 Liposomes

• 7.2.2.2 Polycation Conjugation

• 7.2.2.3 Naked DNA Injection

• 8. TARGETING AND GENE EXPRESSION

• 9. GENERATION OF MOUSE MODELS FOR GENE THERAPY

• 10. CANDIDATES OF INHERITED DISEASES OF THE LUNG

• 10.1 Gene Therapy for Inherited Diseases of Lung

• 10.1.1 Vector System for Gene Delivery

• 10.1.1.1 Viral vectors

• i. Retroviral vectors

• ii. Adenoviral vectors

• iii. Adeno-Associated Virus

• 10.1.1.2 Non-Viral Vectors

• 10.2 Gene Therapy for Cystic Fibrosis

• 11. GENE THERAPY FOR NON-HERITABLE DISEASES

• 11.1 Gene therapy for HIV/AIDS

• 11.1.1 HIV Life Cycle

• 11.1.2 Cellular Vehicles for HIV Gene Therapy

• 11.1.3 Gene Therapy Strategies for HIV/Aids

• 11.1.4 Gene Therapy Based Immunotherapy

• 11.1.5 Direct antiviral transgene

• 11.1.5.1 Interferon

• 11.1.5.2 RNA Strategies

• 11.2 Gene Therapy of Cancer

• 11.2.1 Genetics of Cancer

• 11.2.2 Gene Therapy Strategies for Cancer

• 11.2.2.1 Pro-drug Activation Vectors

• 11.2.2.2 Immunotherapy

• 12. ETHICS OF GENE THERAPY

• 12.1 Ethical Analysis

• 12.2 Application to Somatic Cell Gene Therapy

• 12.3 Somatic Cell Therapies and the Problem of Enhancement Engineering

• 12.4 Germ Line gene Therapy

• 12.5 Fair Distribution of Resources

• 12.6 Recommendations

• 13. CONCLUSIONS

• 14. SUMMARY

• 15. REFERENCES