Kursussøgning, efter- og videreuddannelse – Københavns Universitet

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Kursussøgning, efter- og videreuddannelse

Gene Therapy

Practical information
Study year 2016/2017
Block 3
Programme level Full Degree Master
Course responsibles
  • Anne Nørremølle (7-637070677071674275777066306d7730666d)
  • Niels Erik Møllegaard (7-706b676e75676f4275777066306d7730666d)
  • Department of Cellular and Molecular Medicine
Course number: SMOK14002U

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.

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

Recommended prerequisites

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.


MSc Programme in Molecular Biomedicine
MSc Programme in Biology-Biotechnology


Study Board of Biomolecular Sciences and Technology


Anne Nørremølle
Niels Erik Møllegaard
Thomas Bentin
Peter E. Nielsen


1 block


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Teaching and learning methods

Lectures and discussion classes






Research papers and reviews


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


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

Criteria for exam assessment

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

Censorship form

No external censorship
Several internal examiners/co-examiners

Exam period

Two last weeks of the block.

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