Department of Biomedical Engineering

GBE 440 | Course Introduction and Application Information

Course Name

Gene Therapy

Code	Semester	Theory (hour/week)	Application/Lab (hour/week)	Local Credits	ECTS
GBE 440	SPRING	3	0	3	5

Prerequisites	None
Course Language	English
Course Type	ELECTIVE_COURSE
Course Level	First Cycle
Mode of Delivery	Face-To-Face
Teaching Methods and Techniques of the Course	Discussion Group Work Q&A Lecture / Presentation
National Occupational Classification Code	-
Course Coordinator	Prof. Dr. Zeynep Fırtına Karagonlar
Course Lecturer(s)	Prof. Dr. Zeynep Fırtına Karagonlar
Assistant(s)	-

Course Objectives

The aim of this course is to inform students about viral and non-viral gene transfer techniques used in gene therapy and the molecular basis of these techniques. In this course, gene therapy applications in hereditary or acquired diseases in recent years will be explained and the importance of gene therapy as a treatment option will be discussed.

Learning Outcomes

The students who succeeded in this course;

Name	Description	PC Sub	* Contribution Level
Name	Description	PC Sub	1	2	3	4	5
LO1	Explain the molecular basis of gene therapy approaches	1.2				X
LO2	Identify viral and non-viral vectors used in gene transfer	1.5				X
LO3	Model the structures of viruses used as vectors in gene therapy, and individually report and present this model verbally.	1.5				X
LO4	Discuss gene therapy from an ethical perspective	7.1				X

Course Description

This course covers the basics of gene therapy, viral and non-viral gene delivery techniques, and an explanation of how gene therapy is used as a treatment option.

Related Sustainable Development Goals

Course Category	Core Courses
	Major Area Courses	X
	Supportive Courses
	Media and Managment Skills Courses
	Transferable Skill Courses

WEEKLY SUBJECTS AND RELATED PREPARATION STUDIES

Week	Subjects	Required Materials	Learning Outcome
1	Introduction to gene therapy	A Handbook of Gene and Cell Therapy - Chapter 1	LO1
2	Non-viral vectors in gene therapy	A Handbook of Gene and Cell Therapy - Chapter 2	LO2
3	Viral vectors in gene therapy 1	A Handbook of Gene and Cell Therapy - Chapter 3	LO2
4	Viral vectors in gene therapy 2	A Handbook of Gene and Cell Therapy - Chapter 3	LO2
5	Barriers to gene therapy	A Handbook of Gene and Cell Therapy - Chapter 4	LO1
6	Stem cells and tissue regeneration-1	A Handbook of Gene and Cell Therapy - Chapter 5	LO1
7	Midterm Exam		-
8	Stem cells and tissue regeneration-2	A Handbook of Gene and Cell Therapy - Chapter 5	LO1
9	Gene therapy strategies: gene augmentation	A Handbook of Gene and Cell Therapy - Chapter 6	LO3
10	Gene therapy strategies: gene silencing	A Handbook of Gene and Cell Therapy - Chapter 7	LO3
11	Gene editing	A Handbook of Gene and Cell Therapy - Chapter 8	LO1
12	Gene therapy applications: Preclinical studies and clinical trials	A Handbook of Gene and Cell Therapy - Chapter 9	LO4
13	Gene therapy in cancer	An Introduction to Molecular Medicine and Gene Therapy, Chapter 6-7	LO4
14	Gene Therapy for Ophthalmology and Cardiovascular Diseases	A Handbook of Gene and Cell Therapy - Chapter 9	LO3
15	Gene therapy in neurodegenerative diseases		-
16	Final Exam		-

Course Notes/Textbooks	An Introduction to Molecular Medicine and Gene Therapy 1st Edition by Thomas F. Kresina ISBNs: 0-471-39188-3 (Hardback); 0-471-22387-5 (Electronic)
Suggested Readings/Materials	An Introduction to Human Molecular Genetics (2nd Edition) J.J. Pasternak 2005

EVALUATION SYSTEM

Semester Activities	Number	Weighting	LO1	LO2	LO3	LO4
Quizzes / Studio Critiques	1	10
Homework / Assignments	1	20	X	X
Presentation / Jury	1	20			X	X
Midterm	1	20	X	X	X	X
Final Exam	1	30	X	X	X	X
Total	5	100

ECTS / WORKLOAD TABLE

Semester Activities	Number	Duration (Hours)	Workload
Participation	-	-	-
Theoretical Course Hours	16	3	48
Laboratory / Application Hours	-	-	-
Study Hours Out of Class	16	2	32
Field Work	-	-	-
Quizzes / Studio Critiques	1	5	5
Portfolio	-	-	-
Homework / Assignments	1	10	10
Presentation / Jury	1	15	15
Project	-	-	-
Seminar / Workshop	-	-	-
Oral Exams	-	-	-
Midterms	1	20	20
Final Exam	1	20	20
		Total	150

COURSE LEARNING OUTCOMES AND PROGRAM QUALIFICATIONS RELATIONSHIP

#	PC Sub	Program Competencies/Outcomes	* Contribution Level
#	PC Sub	Program Competencies/Outcomes	1	2	3	4	5
1	Engineering Knowledge: Knowledge of mathematics, science, basic engineering, computation, and related engineering discipline-specific topics; the ability to apply this knowledge to solve complex engineering problems.
	1	Mathematics
	2	Science				LO1
	3	Basic Engineering
	4	Computation
	5	Related engineering discipline-specific topics				LO2 LO3
	6	The ability to apply this knowledge to solve complex engineering problems
2	Problem Analysis: Ability to identify, formulate and analyze complex engineering problems using basic knowledge of science, mathematics and engineering, and considering the UN Sustainable Development Goals relevant to the problem being addressed.
3	Engineering Design: The ability to devise creative solutions to complex engineering problems; the ability to design complex systems, processes, devices or products to meet current and future needs, considering realistic constraints and conditions.
	1	Ability to design creative solutions to complex engineering problems
	2	Ability to design complex systems, processes, devices or products to meet current and future needs, considering realistic constraints and conditions
4	Use of Techniques and Tools: Ability to select and use appropriate techniques, resources, and modern engineering and computing tools, including estimation and modeling, for the analysis and solution of complex engineering problems, while recognizing their limitations.
5	Research and Investigation: Ability to use research methods to investigate complex engineering problems, including literature research, designing and conducting experiments, collecting data, and analyzing and interpreting results.
	1	Literature research for the study of complex engineering problems
	2	Designing experiments
	3	Ability to use research methods, including conducting experiments, collecting data. analyzing and interpreting results
6	Global Impact of Engineering Practices: Knowledge of the impacts of engineering practices on society, health and safety, economy, sustainability, and the environment, within the context of the UN Sustainable Development Goals; awareness of the legal implications of engineering solutions.
	1	Knowledge of the impacts of engineering practices on society, health and safety, economy, sustainability, and the environment, within the context of the UN Sustainable Development Goals
	2	Awareness of the legal implications of engineering solutions
7	Ethical Behavior: Acting in accordance with the principles of the engineering profession, knowledge about ethical responsibility; awareness of being impartial, without discrimination, and being inclusive of diversity.
	1	Acting in accordance with the principles of the engineering profession, knowledge about ethical responsibility ethical responsibility				LO4
	2	Awareness of being impartial and inclusive of diversity, without discriminating on any subject
8	Individual and Teamwork: Ability to work effectively, individually and as a team member or leader on interdisciplinary and multidisciplinary teams (face-to-face, remote or hybrid).
	1	Ability to work individually and within the discipline
	2	Ability to work effectively as a team member or leader in multidisciplinary teams (face-to-face, remote or hybrid)
9	Verbal and Written Communication: Taking into account the various differences of the target audience (such as education, language, profession) on technical issues.
	1	Ability to communicate verbally
	2	Ability to communicate effectively in writing
10	Project Management: Knowledge of business practices such as project management and economic feasibility analysis; awareness of entrepreneurship and innovation.
	1	Knowledge of business practices such as project management and economic feasibility analysis
	2	Awareness of entrepreneurship and innovation
11	Lifelong Learning: Lifelong learning skills that include being able to learn independently and continuously, adapting to new and developing technologies, and thinking questioningly about technological changes.