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10 Modules / ~100 pages
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~25 Modules / ~400 pages

Recent Advances in Gene Therapy Research
( 30 Modules )

Module #1
Introduction to Gene Therapy
Overview of gene therapy, its history, principles, and applications
Module #2
Gene Therapy Vectors:Viral and Non-Viral
Overview of viral and non-viral vectors used in gene therapy, including AAV, Lentivirus, and Lipid nanoparticles
Module #3
Gene Editing Technologies:CRISPR/Cas9 and Beyond
In-depth overview of CRISPR/Cas9 and other gene editing technologies, including base editing and prime editing
Module #4
Gene Regulation and Expression
Mechanisms of gene regulation, including transcriptional and post-transcriptional control, and strategies for gene expression modulation
Module #5
Gene Therapy for Genetic Disorders
Recent advances in gene therapy for inherited diseases, including sickle cell anemia, muscular dystrophy, and cystic fibrosis
Module #6
Cancer Gene Therapy
Strategies for cancer gene therapy, including gene silencing, gene editing, and gene delivery approaches
Module #7
Inherited Retinal Diseases:Gene Therapy and Beyond
Recent advances in gene therapy for inherited retinal diseases, including Leber congenital amaurosis and choroideremia
Module #8
Gene Therapy for Neurological Disorders
Gene therapy approaches for neurological disorders, including Parkinsons disease, Huntingtons disease, and spinal muscular atrophy
Module #9
Gene Therapy for Infectious Diseases
Strategies for gene therapy against infectious diseases, including HIV, tuberculosis, and influenza
Module #10
Gene Therapy Manufacturing and Scale-Up
Challenges and strategies for large-scale manufacturing of gene therapies, including vector production and purification
Module #11
Gene Therapy Regulatory Landscape
Overview of regulatory agencies and guidelines for gene therapy, including FDA, EMA, and WHO
Module #12
Ethical Considerations in Gene Therapy
Ethical implications of gene therapy, including germline editing, gene drives, and access to gene therapy
Module #13
Immune Response and Gene Therapy
Immune responses to gene therapy vectors and strategies for immune modulation
Module #14
Biomarkers and Gene Therapy Monitoring
Development and use of biomarkers for gene therapy monitoring and outcome assessment
Module #15
Gene Therapy Combination Therapies
Strategies for combining gene therapy with other therapeutic modalities, including immunotherapy and small molecules
Module #16
Gene Therapy for Rare Diseases
Gene therapy approaches for rare diseases, including lysosomal storage disorders and neuromuscular disorders
Module #17
Gene Therapy Delivery and Targeting
Strategies for targeted gene therapy delivery, including tissue-specific and cell-specific targeting
Module #18
Gene Therapy Safety and Toxicity
Assessment and mitigation of gene therapy-related safety and toxicity concerns
Module #19
Gene Therapy for Cardiovascular Diseases
Gene therapy approaches for cardiovascular diseases, including heart failure and atherosclerosis
Module #20
Gene Therapy for Respiratory Diseases
Gene therapy approaches for respiratory diseases, including cystic fibrosis and alpha-1 antitrypsin deficiency
Module #21
Gene Therapy for Gastrointestinal Diseases
Gene therapy approaches for gastrointestinal diseases, including inflammatory bowel disease and metabolic disorders
Module #22
Gene Therapy for Skin Diseases
Gene therapy approaches for skin diseases, including epidermolysis bullosa and ichthyosis
Module #23
Gene Therapy for Musculoskeletal Diseases
Gene therapy approaches for musculoskeletal diseases, including osteoarthritis and muscular dystrophy
Module #24
Gene Therapy for Ocular Diseases
Gene therapy approaches for ocular diseases, including age-related macular degeneration and glaucoma
Module #25
Gene Therapy for Auditory Diseases
Gene therapy approaches for auditory diseases, including sensorineural hearing loss and tinnitus
Module #26
Gene Therapy for Hematological Disorders
Gene therapy approaches for hematological disorders, including sickle cell disease and β-thalassemia
Module #27
Gene Therapy for Kidney Diseases
Gene therapy approaches for kidney diseases, including chronic kidney disease and polycystic kidney disease
Module #28
Gene Therapy for Liver Diseases
Gene therapy approaches for liver diseases, including inherited liver diseases and liver cancer
Module #29
Gene Therapy for Endocrine Disorders
Gene therapy approaches for endocrine disorders, including diabetes and thyroid diseases
Module #30
Course Wrap-Up & Conclusion
Planning next steps in Recent Advances in Gene Therapy Research career


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