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Swedish title Cell och genterapi
English title Cell and Gene Therapy
Course number 2444
Credits 1.5
Responsible KI department Institutionen för medicin, Huddinge
Specific entry requirements No specific entry requirements
Grading Passed /Not passed
Established by The Committee for Doctoral Education
Established 2019-09-16
Purpose of the course Gene Therapy is a growing field with applications both in research and development as well as a therapeutic tool for patients with cancer or inherited genetic disorders. The purpose of the course is to deepen the participant’s knowledge in gene-therapeutic tools and its applications in disease. The course will focus on genetic modification of human cells and their application in cancer and will give an overview of gene-therapeutic options including viral vector production, - transduction and CRISPR technology. Furthermore, we will touch upon ethical aspects of gene therapy.
Intended learning outcomes After the course, the student should be able to;
1. Explain mechanisms that are involved in viral gene integration in mammalian cells.
2. Apply their knowledge in molecular biology to genetic modification of mammalian cells. This means they should be able to develop cloning strategies for a gene of interest into a viral vector and propose a strategy for in vivo genetic manipulation.
3. Understand the application of CAR T and CAR NK cells
4. Apply the knowledge in cancer or single gene disorders and develop treatment schemes for patients with these disorders using gene therapy
5. Understand the regulations concerning cell and gene therapy
6. Understand the strengths and drawback of current clinical trials
Contents of the course The first part of the course will cover the basic aspects of gene regulation, cell culture, and gene transfer methods. In addition, the recent advances in vector development will be discussed. The second part of the course will deal with studies performed with human cells, with special emphasis on the hematology field.
Teaching and learning activities The pedagogic learning activities on the course consist of lectures, group discussions and student presentations.
Compulsory elements All lectures and teaching activities are mandatory. Absence from mandatory parts of the course will have to be compensated by other relevant activities after discussion with the course leaders.
Examination Oral group presentation and individual assignment based on a case study. Every student will be individually assessed.
Literature and other teaching material Students will be informed about relevant literature prior to course start.
Course responsible Evren Alici
Institutionen för medicin, Huddinge

073-6261412
Evren.Alici@ki.se

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