SMOK14002U Gene Therapy
Gene Therapy encompasses a collection of techniques that use genetic material to manipulate the genome of a patient for the treatment of inherited or acquired diseases. It is a field that currently undergoes immense development, and new principles and techniques are constantly emerging. The course builds on and merges the fields of genetics, biochemistry and molecular biology, and will include clinical aspects of gene therapy.
This course covers a range of methods and basic cell biology concepts relevant to gene therapy, and related applications, including genome editing methods (Zinc Finger Nucleases, TAL effectors, CRISPR/Cas), RNA interference (siRNAs, microRNAs), antisense, antigene, ribozymes and DNA vaccines. The course will address challenges encountered in gene therapy, such as agent delivery, off-target effects, cell toxicity and immune response induction. The course is taught by scientist working in gene therapy related research and development from universities and hospitals as well as from industry. Course literature consists of recent research papers and reviews.
Competencies
After the course the student should be able to:
- understand the present opportunities, problems and limitations in gene therapy
- independently formulate minor projects aiming at a specific gene therapeutical effect
- apply theoretical and technical knowledge in understanding and evaluating new principles in gene therapy
Skills
After the course the student should be able to:
- critically evaluate projects in gene therapy
- choose, apply and evaluate techniques aiming at regulation or editing of genes and gene expression
Knowledge
After the course the student should know:
- the theoretical background for gene therapy and the effect of alterations in gene expression
- the most important principles and technologies applied in gene therapy
- the necessary control experiments applied in gene therapy development
Research papers and reviews
- Category
- Hours
- Class Instruction
- 25
- E-Learning
- 10
- Exam Preparation
- 66
- Lectures
- 25
- Preparation
- 80
- Total
- 206
- Credit
- 7,5 ECTS
- Type of assessment
- Written assignment, 2 weeksWritten assignment: Formulation of a research proposal designing and critically evaluating experiments aiming at answering a specific question related to gene therapy.
- Aid
- All aids allowed
- Marking scale
- 7-point grading scale
- Censorship form
- No external censorship
- Exam period
- Two last weeks of the block.
- Re-exam
- Oral exam
Criteria for exam assesment
The paper produced during exam will be evaluated using the criteria mentioned in the learning outcome.
Course information
- Language
- English
- Course code
- SMOK14002U
- Credit
- 7,5 ECTS
- Level
- Full Degree Master
- Duration
- 1 block
- Placement
- Block 3
- Schedule
- A1 (Tues 8-10 + Thurs 8-12) And A2 (Tues 10-12 + Thurs 13-17)
- Course capacity
- 50
- Study board
- Study Board of Biomolecular Sciences and Technology
Contracting department
- Department of Cellular and Molecular Medicine
Course responsibles
- Niels Erik Møllegaard (7-827d79808779815487898278427f8942787f)
- Anne Nørremølle (7-758282798283795487898278427f8942787f)
Lecturers
Niels Erik Møllegaard
Anne Nørremølle
Thomas Bentin
Lis Hasholt
Peter E. Nielsen
m.fl.