1. |
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Genetic Pharmacology I |
1. Antigene Strategy
2. Antisense Strategy |
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Genetic Pharmacology II |
2. Antigene Strategy
3. Delivery Issues |
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2. |
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Principles of RNAi |
1. Principles of RNAi Trigger Expression for Gene Therapy |
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Principles of RNAi |
2. On Demand Alternative Splicing for Gene Rescue
3. Nuclease-Mediated Targeted Genetic Correction |
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3. |
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Small regulatory RNAs |
1. Therapeutic agents based on
ribonucleic acid
2. small interfering RNAs (siRNAs) |
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Small regulatory RNAs |
3. microRNAs mimics (miRNA mimics) |
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4. |
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Gene Delivery |
1. Cellular varriers to gene delivery
2. Endocytosis |
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Gene Delivery |
3. Escaped from the intracellular vesicle compartment
4. Direct inoculation of DNA and RNA
5. Sonoporation, microparticles, Liposomes, cationic polymers |
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5. |
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CRISPR/Cas9 Genome Engineering |
1. Origin of CRISPR/Cas system
2. Application of CRISPR/Cas system
3. Major components of CRISPR/Cas9 genome editing system
4. Mechanism of CRISPR/Cas9 genome editing
5. Delivery of CRISPR/Cas9
6. Advantages of CRISPR/Cas9 genome editing |
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Vectors and Gene Delivery Techniques 1 |
1. Vectors (Viral vector)
2. Retrovirus- and Lentivirus-Derived Vectors for Gene Transfer and Therapy |
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6. |
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Vectors and Gene Delivery Techniques 1 |
2. γ-Retrovirus- and Lentivirus-Derived Vectors for Gene Transfer and Therapy
3. Adenovirus vectors |
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Vectors and Gene Delivery Techniques 1 |
3. Adenovirus, Adeno-Associated Virus (AAV) Vectors
4. Herpes Simplex Virus (HSV-1)-Based Vectors: Applications for Gene Transfer, Gene Therapy, Cancer Virotherapy and Vaccination |
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7. |
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Vectors and Gene Delivery Techniques 2 |
1. Vectors (Non-viral vector)
2. Macromolecular Conjugates for Non-Viral Nucleic Acid Delivery
3. Auto-Associative Lipid-Based Systems for Non-Viral Nucleic Acid Delivery
4. Electrotransfer/Electroporation for Non-Viral Nucleic Acid Delivery |
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8. |
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Therapeutic Applications 1 |
1. Oncolytic Adenoviruses for Cancer Gene Therapy |
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9. |
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Therapeutic Applications 1 |
2. Progress in DNA Vaccine Approaches for Cancer Immunotherapy |
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10. |
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Therapeutic Applications 2 |
1. Gene Therapy of the Beta-Hemoglobinopathies
2. Gene Therapy for Hemophilia A and B |
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Therapeutic Applications 2 |
3. Experimental and Clinical Ocular Gene Therapy
4. Gene Therapy of Neurological Diseases |
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11. |
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Gene Vector Production |
1. Production and Purification of Viral Vectors |
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Gene Vector Production |
2. Safety Considerations Related to Gene Vector Production |
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12. |
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Clinical application of gene therapy: General considerations |
1. Number/ phases of clinical trials
2. Disease addressed by gene therapy clinical trials
3. Therapeutic genes used in the clinical trials
4. Modality of therapeutic gene delivery in gene therapy clinical trials |
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13. |
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Ethical and social problems of gene therapy |
1. Safety of clinical experimentation
2. Ethical and social problems of gene therapy
3. Regulation and guidelines of gene therapy |
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