Gene Editing

  1. Therapeutic Applications
    1. Clinical Gene Editing Approaches
      1. Somatic Gene Editing
        1. Definition and Scope
          1. Non-heritable Modifications
            1. Therapeutic Applications
            2. Germline Gene Editing
              1. Definition and Implications
                1. Heritable Modifications
                  1. Ethical and Regulatory Considerations
                  2. Clinical Trial Design
                    1. Phase I Safety Studies
                      1. Efficacy Endpoints
                        1. Patient Selection Criteria
                      2. Ex Vivo Gene Therapy
                        1. Cell Modification Strategies
                          1. Hematopoietic Stem Cell Editing
                            1. HSC Isolation and Culture
                              1. Editing Protocols
                                1. Engraftment Considerations
                                2. T Cell Engineering
                                  1. CAR-T Cell Development
                                    1. TCR Engineering
                                      1. Checkpoint Inhibition
                                    2. Clinical Applications
                                      1. Sickle Cell Disease
                                        1. HbF Induction Strategies
                                          1. BCL11A Targeting
                                            1. Clinical Trial Results
                                            2. Beta-Thalassemia
                                              1. HbF Reactivation
                                                1. Treatment Outcomes
                                                2. Cancer Immunotherapy
                                                  1. CAR-T Cell Therapy
                                                    1. Tumor-Infiltrating Lymphocytes
                                                    2. Primary Immunodeficiencies
                                                      1. SCID Treatment
                                                        1. CGD Correction
                                                      2. Manufacturing and Quality Control
                                                        1. GMP Production
                                                          1. Quality Assurance
                                                            1. Product Characterization
                                                          2. In Vivo Gene Therapy
                                                            1. Direct Delivery Approaches
                                                              1. Systemic Administration
                                                                1. Local Injection
                                                                  1. Organ-specific Delivery
                                                                  2. Target Diseases
                                                                    1. Inherited Blindness
                                                                      1. Leber Congenital Amaurosis
                                                                        1. Retinal Dystrophies
                                                                        2. Muscular Disorders
                                                                          1. Duchenne Muscular Dystrophy
                                                                            1. Myotonic Dystrophy
                                                                            2. Metabolic Diseases
                                                                              1. Hereditary Transthyretin Amyloidosis
                                                                                1. Glycogen Storage Diseases
                                                                              2. Delivery Challenges
                                                                                1. Tissue Penetration
                                                                                  1. Immune Responses
                                                                                    1. Dosing Optimization
                                                                                      1. Safety Monitoring
                                                                                    2. Emerging Therapeutic Areas
                                                                                      1. Neurological Disorders
                                                                                        1. Huntington's Disease
                                                                                          1. Amyotrophic Lateral Sclerosis
                                                                                            1. Alzheimer's Disease
                                                                                            2. Infectious Diseases
                                                                                              1. HIV Cure Strategies
                                                                                                1. Antiviral Approaches

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                                                                                            8. Applications in Biomedical Research

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                                                                                            10. Applications in Agriculture and Biotechnology