Gene Therapy

Course content

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.


MSc Programme in Biotechnology
MSc Programme in Molecular Biomedicine

Learning outcome


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



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



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

Lectures and discussion classes

Research papers and reviews

The students should have practical laboratory and research experiences.

Open to students with a Bachelor degree in Molecular Biomedicine, Biology-Biotechnology, Biochemistry and Biology and others who have similar knowledge.

Continuous feedback during the course of the semester

Spørgsmåls og diskussionstimer i løbet af kurset = mundtlig, individuel, kollektiv og løbende feedback

Mundtlig feedback på fremlæggelser

7,5 ECTS
Type of assessment
Written assignment, 2 weeks
Written assignment: Formulation of a research proposal designing and critically evaluating experiments aiming at answering a specific question related to gene therapy.
All aids allowed
Marking scale
7-point grading scale
Censorship form
No external censorship
Several internal examiners/co-examiners
Criteria for exam assessment

The paper produced during exam will be evaluated using the criteria mentioned in the learning outcome.

  • Category
  • Hours
  • Lectures
  • 25
  • Class Instruction
  • 25
  • Preparation
  • 80
  • E-Learning
  • 10
  • Exam Preparation
  • 66
  • English
  • 206