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| MBGL5035 | Gene therapy and genome editing | 3+0+0 | ECTS:7.5 | | Year / Semester | Fall Semester | | Level of Course | Second Cycle | | Status | Elective | | Department | DEPARTMENT of MOLECULAR BIOLOGY and GENETICS | | Prerequisites and co-requisites | None | | Mode of Delivery | Face to face | | Contact Hours | 14 weeks - 3 hours of lectures per week | | Lecturer | Dr. Öğr. Üyesi Cihan İNAN | | Co-Lecturer | | | Language of instruction | Turkish | | Professional practise ( internship ) | None | | | | The aim of the course: | | Along with developing technologies, cellular and personalized methods have begun to appear in the treatment of diseases. In this course it is aimed to educate students about endonucleases and gene engineering applications especially their roles in treatment of diseases. |
| Programme Outcomes | CTPO | TOA | | Upon successful completion of the course, the students will be able to : | | | | PO - 1 : | Know why we need gene regulation. | 2,3,6,7 | 1 | | PO - 2 : | Can compare gene regulation methods and their superiority with each other | 2,3,6,7 | 1,3 | | PO - 3 : | Explores current gene therapy practice examples | 2,3,6,7 | 1,3,5 | | CTPO : Contribution to programme outcomes, TOA :Type of assessment (1: written exam, 2: Oral exam, 3: Homework assignment, 4: Laboratory exercise/exam, 5: Seminar / presentation, 6: Term paper), PO : Learning Outcome | | |
| Examination of past and current methods of gene therapy and genome editing. Use of zinc finger nuclases, TALENs and CRISPR / Cas9 nuclease methods. Genetic applications in diseases, gene engineering applications, call models, current working examples. |
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| Course Syllabus | | Week | Subject | Related Notes / Files | | Week 1 | Introduction to gene therapy. History of gene editing | | | Week 2 | What is gene therapy? What are the types of gene therapy? What are Gene Therapy Strategies? | | | Week 3 | Choice of the Therapeutic Target, Apoptosis - Necrosis - Autophagy. Administration Routes. Delivery Systems. Expression and Persistence of the Therapy. | | | Week 4 | The Development and Use of Zinc-Finger Nucleases. The Use and Development of TAL Effector Nucleases | | | Week 5 | The Use and Development of CRISPR method | | | Week 6 | The Use and Development of CRISPR method | | | Week 7 | Non-viral vectors in gene therapy | | | Week 8 | Mid-term exam | | | Week 9 | Non-viral vectors in gene therapy | | | Week 10 | Viral vectors in gene therapy | | | Week 11 | Viral vectors in gene therapy | | | Week 12 | Barriers to Gene Transfer | | | Week 13 | Gene Therapy Strategies: Gen addition | | | Week 14 | Gene Therapy Strategies: Gen silencing | | | Week 15 | Current gene therapy and genome editing examples | | | Week 16 | Final exam | | | |
| 1 | Nobrega, C., Mendonca, L., Matos, C.A., A handbook of Gene and Cell Therapy, 2020, Springer | | | |
| 1 | Cathomen, T., Hirsch, M., Porteus, M., Genome editing. The next step in gene therapy, 2016, Springer | | | |
| Method of Assessment | | Type of assessment | Week No | Date | Duration (hours) | Weight (%) | | Mid-term exam | 8 | | 1 | 30 | | Presentation | 15 | | 1 | 20 | | End-of-term exam | 16 | | 1 | 50 | | |
| Student Work Load and its Distribution | | Type of work | Duration (hours pw) | No of weeks / Number of activity | Hours in total per term | | Yüz yüze eğitim | 3 | 14 | 42 | | Sınıf dışı çalışma | 6 | 16 | 96 | | Laboratuar çalışması | 0 | 0 | 0 | | Arasınav için hazırlık | 4 | 8 | 32 | | Arasınav | 1 | 1 | 1 | | Uygulama | 0 | 0 | 0 | | Klinik Uygulama | 0 | 0 | 0 | | Ödev | 1 | 2 | 2 | | Proje | 0 | 0 | 0 | | Kısa sınav | 0 | 0 | 0 | | Dönem sonu sınavı için hazırlık | 3 | 16 | 48 | | Dönem sonu sınavı | 1 | 1 | 1 | | Total work load | | | 222 |
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