Stem Cell And Tissue Engineering (DR) Preview
PhD	TR-NQF-HE: Level 8	QF-EHEA: Third Cycle	EQF-LLL: Level 8

Course Introduction and Application Information

Course Code:

KHDD6206

Course Name:

Advanced Gene Therapy and Applications

Semester:

Fall
Spring

Course Credits:

ECTS

Language of instruction:

Turkish

Course Condition:

Does the Course Require Work Experience?:

Type of course:

Departmental Elective

Course Level:

PhD

TR-NQF-HE:8. Master`s Degree

QF-EHEA:Third Cycle

EQF-LLL:8. Master`s Degree

Mode of Delivery:

Face to face

Course Coordinator:

Dr. Öğr. Üy. AYŞE KÖYLÜ

Course Lecturer(s):

Ayca İLTER

Course Assistants:

Course Objective and Content

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

Learning Outcomes

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

Course Flow Plan

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

Sources

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 - Program Learning Outcome Relationship

Course Learning Outcomes	1	2	3	4
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.

Course - Learning Outcome Relationship

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

Assessment & Grading

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

Workload and ECTS Credit Calculation

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