After completion of this course, the student will have obtained fundamental knowledge about molecular mechanisms of novel therapeutics targeting DNA/RNA and proteins. Moreover, the student will be able to appraise the steps involved in the development and delivery of (personalized) therapeutics.
More specifically, the student will be able to:
- appreciate the importance of drug design in optimizing efficacy, potency and safety of therapeutics.
- relate different mechanisms of drug targeting and delivery to the needs of a particular disease.
- describe current genetic therapy approaches to inherited diseases.
- apply the knowledge of molecular pathways of signal transduction to the understanding of the working mechanism of novel medicines in the fields of cancer and pain.
- justify the steps involved in drug development: from target discovery, lead optimization and preclinical studies to clinical trials.
- Implement the obtained knowledge to write a proposal on a novel treatment option for a particular disease.
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This course will introduce students into novel therapeutics applied to treat diseases that pose a heavy burden on society, i.e. (lung) cancer and (chronic) pain and inherited retinal diseases. Since the development of new therapeutics is increasingly based on profound fundamental knowledge, the molecular mechanisms of these medicines will be thoroughly discussed. Moreover, current techniques in drug design, targeting and delivery will be discussed as well as the problems caused by adverse effects and the occurrence of resistance against certain types of medicine.
Students will be introduced into the subjects by means of lectures. Optional tutorials will be organized in order to enable the students to refresh their basic knowledge on key signal transduction pathways. Team-based learning sessions will allow students to apply the knowledge they acquired. In order to write a proposal on the design of a potential treatment option for an inherited retinal disease, students will be stimulated to mine current literature. This course will be given in close collaboration with preclinical investigators and clinical professionals that have experience with the therapeutics discussed.
Subjects
- Drug development pipeline
- Types of (pharmacological) interventions
- Drug design
- Drug targeting and delivery
- Personalized medicine
- Adverse effects and resistance
- DNA/RNA-based therapy
- Protein-targeted therapy: small molecules and biologics
- Signaling (through GPCRs and RTKs) in health and disease
- Targeting inherited retinal diseases
- Targeting (chronic) pain
- Targeting (lung) cancer
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