SMOK14002U Gene Therapy

Volume 2024/2025

MSc Programme in Biotechnology - restricted elective
MSc Programme in Molecular Biomedicine - restricted elective


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.

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

Research papers and reviews

The students should have practical laboratory and research experiences.
Lectures and discussion classes
Open to students with a Bachelor degree in Molecular Biomedicine, Biology-Biotechnology, Biochemistry and Biology and others who have similar knowledge.
  • Category
  • Hours
  • Lectures
  • 25
  • Class Instruction
  • 25
  • Preparation
  • 80
  • E-Learning
  • 10
  • Exam Preparation
  • 66
  • Total
  • 206
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
Type of assessment details
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
Exam period

Two last weeks of the block.

Criteria for exam assesment

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