Medical Genetics

  1. Gene Therapy and Novel Therapeutics
    1. Principles of Gene Therapy
      1. Viral Vectors
        1. Adenoviral Vectors
          1. High Transduction Efficiency
            1. Immunogenic Response
              1. Episomal Expression
              2. Lentiviral Vectors
                1. Stable Integration
                  1. Dividing and Non-dividing Cells
                    1. HIV-Based System
                    2. Adeno-Associated Viral Vectors
                      1. Low Immunogenicity
                        1. Tissue Tropism
                          1. Episomal Persistence
                        2. Non-viral Vectors
                          1. Liposomes
                            1. Lipid-Based Delivery
                              1. Reduced Immunogenicity
                                1. Lower Efficiency
                                2. Naked DNA
                                  1. Direct Injection
                                    1. Electroporation
                                      1. Limited Uptake
                                    2. In vivo Approaches
                                      1. Direct Delivery
                                        1. Systemic Administration
                                          1. Tissue-Specific Targeting
                                          2. Ex vivo Approaches
                                            1. Cell Modification
                                              1. Reinfusion
                                                1. Reduced Systemic Exposure
                                              2. Therapeutic Strategies
                                                1. Gene Replacement
                                                  1. Functional Gene Addition
                                                    1. Recessive Disorders
                                                      1. Viral Vector Delivery
                                                      2. Gene Silencing
                                                        1. RNA Interference
                                                          1. siRNA Mechanism
                                                            1. miRNA Pathway
                                                              1. Therapeutic Applications
                                                              2. Antisense Oligonucleotides
                                                                1. Complementary Binding
                                                                  1. RNase H Activation
                                                                    1. Splice Modulation
                                                                  2. Genome Editing
                                                                    1. CRISPR-Cas9
                                                                      1. Guide RNA Design
                                                                        1. Cas9 Nuclease
                                                                          1. Homology-Directed Repair
                                                                            1. Non-Homologous End Joining
                                                                            2. TALENs
                                                                              1. Transcription Activator-Like Effectors
                                                                                1. Nuclease Fusion
                                                                                  1. Target Specificity
                                                                                  2. Zinc Finger Nucleases
                                                                                    1. Zinc Finger Domains
                                                                                      1. FokI Nuclease
                                                                                        1. Custom Design
                                                                                      2. Base Editing and Prime Editing
                                                                                        1. Cytosine Base Editors
                                                                                          1. Adenine Base Editors
                                                                                            1. Prime Editing System
                                                                                              1. Reduced Double-Strand Breaks
                                                                                            2. Clinical Applications and Challenges
                                                                                              1. Approved Gene Therapies
                                                                                                1. FDA-Approved Products
                                                                                                  1. Clinical Indications
                                                                                                    1. Treatment Outcomes
                                                                                                    2. Safety and Efficacy
                                                                                                      1. Clinical Trial Design
                                                                                                        1. Endpoint Assessment
                                                                                                          1. Long-term Follow-up
                                                                                                          2. Immune Responses
                                                                                                            1. Vector Immunogenicity
                                                                                                              1. Transgene Immunogenicity
                                                                                                                1. Immunosuppression Strategies
                                                                                                                2. Ethical and Regulatory Issues
                                                                                                                  1. Risk-Benefit Assessment
                                                                                                                    1. Regulatory Approval Process
                                                                                                                    2. Long-Term Follow-Up
                                                                                                                      1. Safety Monitoring
                                                                                                                        1. Efficacy Assessment
                                                                                                                          1. Registry Studies

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                                                                                                                      6. Specific Areas in Medical Genetics

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                                                                                                                      8. Ethical, Legal, and Social Implications