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From Molecular Pathogenesis to Targeted Therapy


Admission requirements

No previous participation in the (strongly overlapping) course “From Signal Transduction to Targeted Therapy”
Basic understanding of signal transduction mechanisms at a level equivalent to the Leiden biomedical sciences bachelor courses ‘Cellular Communication’, and ‘Molecular Biology and Oncology’. Successful completion of How To Write A Research Proposal is strongly recommended.

During this intense course the students will be challenged.


Period: October 20 – November 14, 2014

Regarding the molecular basis of human diseases in particular disorders associated with misregulated TGF-beta family signaling will be studied, including cancer, fibrosis, osteoporosis, and pulmonary hypertention. In addition, identification and functional characterization of novel emerging diagnostic and therapeutic approaches will be discussed.

week 1:
An introduction into the basic concepts of signal transduction and pathophysiological consequences of deregulated signalling, including lectures, an interactive tutorial, and literature presentations and discussions by students.

week 2:
State of the art lectures on signaling, molecular pathogenesis and targeted therapy
by experts in the field (in combination with a PhD course).

(preliminary) program
day 1 Signaling pathways
day 2 Diabetes, ageing and bone-related diseases
day 3 Growth control, stem cells and cancer
day 4 Personalized medicine, targeted therapy and imaging
day 5 presentations by PhD students

week 3 and 4:
Training in computational modeling of a signal transduction pathway, and writing of a report in the form of a grant proposal in which a specific signalling molecule or pathway is proposed for (personalized) drug/clinical development. This will be done under guidance of a mentor that will meet with the students every day. At the end of the course each group will give a presentation on their proposal in a mini-symposium.

In addition, the students get lectures on different aspects of translational research (drug discovery and development, molecular imaging, clinical trials). Visits will be made to a laboratory that participates in a clinical trial and to a biotechnology companies.

This course will in particular train the students in:
Research competences:
Integrate different biomedical disciplines, recording, organizing and analyzing data, choosing appropriate techniques.

Professional competences:
Commitment, motivation and drive, collaborating with peers, respecting the rules of the group. Ability to multi-task (work at several assignments, different topics during the course).


To learn how cells communicate with each other, how (molecular) signals are transduced between cells and into cells, how these processes regulate cellular functions, how misregulated signal transduction events lead to diseases, and how insights in these processes can be translated into targeted therapies providing personalized and more effective treatments for patients with less side effects.

Mode of instruction

Lectures, self-study, work groups.
Pro-active behaviour is expected from the student.

Assessment method

Written reports, oral presentations, student behaviour.

Recommended prior knowledge

  • Alberts B. et al, Molecular Biology of the Cell, 4th ed. 2002 New York: Garland. Chapter 15.

  • Weinberg RA, The Biology of Cancer, Garland Science, Taylor & Francis Group, LLC. Chapters 5 and 6.