Stem Cell And Tissue Engineering (DR) | |||||
PhD | TR-NQF-HE: Level 8 | QF-EHEA: Third Cycle | EQF-LLL: Level 8 |
Course Code: | KHDD6206 | ||||
Course Name: | Advanced Gene Therapy and Applications | ||||
Semester: |
Fall Spring |
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Course Credits: |
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Language of instruction: | Turkish | ||||
Course Condition: | |||||
Does the Course Require Work Experience?: | No | ||||
Type of course: | Departmental Elective | ||||
Course Level: |
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Mode of Delivery: | Face to face | ||||
Course Coordinator: | Dr. Öğr. Üy. AYŞE KÖYLÜ | ||||
Course Lecturer(s): | Ayca İLTER | ||||
Course Assistants: |
Course Objectives: | The aim of this course is to give detailed information about the use of cellular therapies developed with new generation genome modification methods in diseases, the aspects to be developed in these approaches, the advantages and disadvantages of use, and the place of these therapies in the future. |
Course Content: | Gene modification tools, synthetic gene design, introduction to CRISPR-Cas systems, the use of different Cas proteins in gene therapy, the use of Talen and Zinc finger nucleases in gene therapy (advantages and disadvantages), gene transfer methods (viral and non-viral methods), large scale vector production systems in gene therapy, use of genetically modified cells in various diseases and applications that need to be developed |
The students who have succeeded in this course;
1) Learns about different genome modification methods 2) Learns the gene delivery methods and their limitations in gene editing, and how to develop a safe and efficient gene delivery method 3) Gains knowledge about the large-scale production of vectors used in genome modification, its challenges and validations 4) Learns the use of new generation genetically engineered cells in the treatment of diseases and be able to give comments on the aspects that need to be developed |
Week | Subject | Related Preparation |
1) | Introduction to Genome modification: Tools of genetic engineering and synthetic gene design | |
2) | Gene Editing Methods-I: Introduction to CRISPR-Cas systems | |
3) | Gene Editing Methods-II: Zinc Finger nuclease and Talen | |
4) | Gene Delivery Methods-I: Non-viral methods | |
5) | Gen Aktarım Yöntemleri-II: Viral yöntemler | |
6) | Large scale manufacturing systems in gene therapy vector production | |
7) | Midterm exam | |
8) | Use of genetically modified immune system cells in cancer treatment (Chimeric Antigen Receptor T cell Therapy) | |
9) | Gene therapy methods used in HIV (human immunodeficiency virus) treatment | |
10) | Gene therapy methods used in Duchenne Muscular Dystrophy (DMD) treatment | |
11) | Gene therapy methods used in Sickle cell disease | |
12) | Gene Therapy Methods used in Spinal Muscular Atrophy (SMA) | |
13) | Student presentations | |
14) | Student presentations | |
15) | Final exam |
Course Notes / Textbooks: | Dersin kaynak kitabı bulunmamaktadır. / The course does not have a mandatory resource. |
References: | Ders notları ve gen terapi ile ilgili yeni çıkan makaleler/Lecture notes and recent papers related to gene therapy |
Course Learning Outcomes | 1 |
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Program Outcomes | ||||||||||||||||
1) To be able to develop and deepen current and advanced knowledge in the field with original thought and / or research at the level of expertise based on master's qualifications and to reach original definitions that will bring innovation to the field. | ||||||||||||||||
2) To be able to comprehend the interdisciplinary interaction that the field is related to; Ability to reach original results by using knowledge that requires expertise in analyzing, synthesizing and evaluating new and complex ideas. | ||||||||||||||||
3) To be able to evaluate and use new knowledge in the field with a systematic approach. | ||||||||||||||||
4) To be able to develop a new idea, method, design and/or application that brings innovation to the field, or to apply a known idea, method, design and/or application to a different field, to research, comprehend, design, adapt and apply an original subject. | ||||||||||||||||
5) To be able to critically analyze, synthesize and evaluate new and complex ideas. | ||||||||||||||||
6) Gaining high-level skills in using research methods in studies related to the field. | ||||||||||||||||
7) Being able to contribute to the progress in the field by independently carrying out an original work that brings innovations to the field, develops a new idea, method, design and / or application or applies a known idea, method, design and / or application to a different field. | ||||||||||||||||
8) To be able to expand the limits of knowledge in the field by publishing at least one scientific article related to the field in national and/or international refereed journals and/or by producing or interpreting an original work. | ||||||||||||||||
9) Ability to lead in environments that require the resolution of unique and interdisciplinary problems. | ||||||||||||||||
10) To be able to develop new thoughts and methods in the field by using high-level mental processes such as creative and critical thinking, problem solving and decision making. | ||||||||||||||||
11) To be able to critically examine and develop social relations and the norms that guide these relations, and to manage actions to change them when necessary. | ||||||||||||||||
12) To be able to defend their original views in the discussion of the issues in the field with experts and to establish an effective communication showing their competence in the field. | ||||||||||||||||
13) To be able to communicate and discuss at an advanced level in written, oral and visual using a foreign language at least at the C1 General Level of the European Language Portfolio. | ||||||||||||||||
14) To be able to contribute to the process of becoming an information society and maintaining it by introducing scientific, technological, social or cultural advances in the field. | ||||||||||||||||
15) To be able to interact functionally by using strategic decision-making processes in solving the problems encountered in the field. | ||||||||||||||||
16) To be able to contribute to the solution of social, scientific, cultural and ethical problems encountered in the fields related to the field and to support the development of these values. |
No Effect | 1 Lowest | 2 Average | 3 Highest |
Program Outcomes | Level of Contribution | |
1) | To be able to develop and deepen current and advanced knowledge in the field with original thought and / or research at the level of expertise based on master's qualifications and to reach original definitions that will bring innovation to the field. | 3 |
2) | To be able to comprehend the interdisciplinary interaction that the field is related to; Ability to reach original results by using knowledge that requires expertise in analyzing, synthesizing and evaluating new and complex ideas. | 3 |
3) | To be able to evaluate and use new knowledge in the field with a systematic approach. | 3 |
4) | To be able to develop a new idea, method, design and/or application that brings innovation to the field, or to apply a known idea, method, design and/or application to a different field, to research, comprehend, design, adapt and apply an original subject. | 3 |
5) | To be able to critically analyze, synthesize and evaluate new and complex ideas. | 3 |
6) | Gaining high-level skills in using research methods in studies related to the field. | 3 |
7) | Being able to contribute to the progress in the field by independently carrying out an original work that brings innovations to the field, develops a new idea, method, design and / or application or applies a known idea, method, design and / or application to a different field. | 3 |
8) | To be able to expand the limits of knowledge in the field by publishing at least one scientific article related to the field in national and/or international refereed journals and/or by producing or interpreting an original work. | 3 |
9) | Ability to lead in environments that require the resolution of unique and interdisciplinary problems. | 3 |
10) | To be able to develop new thoughts and methods in the field by using high-level mental processes such as creative and critical thinking, problem solving and decision making. | 3 |
11) | To be able to critically examine and develop social relations and the norms that guide these relations, and to manage actions to change them when necessary. | 3 |
12) | To be able to defend their original views in the discussion of the issues in the field with experts and to establish an effective communication showing their competence in the field. | 3 |
13) | To be able to communicate and discuss at an advanced level in written, oral and visual using a foreign language at least at the C1 General Level of the European Language Portfolio. | 3 |
14) | To be able to contribute to the process of becoming an information society and maintaining it by introducing scientific, technological, social or cultural advances in the field. | 3 |
15) | To be able to interact functionally by using strategic decision-making processes in solving the problems encountered in the field. | 3 |
16) | To be able to contribute to the solution of social, scientific, cultural and ethical problems encountered in the fields related to the field and to support the development of these values. | 3 |
Semester Requirements | Number of Activities | Level of Contribution |
Midterms | 1 | % 40 |
Final | 1 | % 60 |
total | % 100 | |
PERCENTAGE OF SEMESTER WORK | % 40 | |
PERCENTAGE OF FINAL WORK | % 60 | |
total | % 100 |
Activities | Number of Activities | Preparation for the Activity | Spent for the Activity Itself | Completing the Activity Requirements | Workload | ||
Course Hours | 2 | 0 | 1 | 15 | 32 | ||
Midterms | 1 | 2 | 2 | 1 | 5 | ||
Final | 1 | 2 | 2 | 1 | 5 | ||
Total Workload | 42 |