Translational Tools II – New Methods

Course content

The main themes covered in the course include:

  • Stem cells, induced pluripotent stem cells, and their applications in disease modeling
  • Precision nuclease-based genome editing
  • Proteolysis targeting chimera (PROTACs)
  • Mass spectrometry for understanding drug mechanisms
  • High resolution microscopy
  • Cryo-EM

BRIDGE - Translational Excellence Programme


Learning outcome

On completion of the course, the participants should be able to:


  • Explain the rationale for the utilization of several advanced research methodologies to characterize protein mechanisms of action in vivo with a focus on two general areas: 

                1) gene editing

                2) cryo-EM

  • Discuss the applications of mass spectrometry-based proteomic approaches for drug target discovery



  • Culture and use mouse embryonic stem cells
  • Mass spectrometry sample preparation for identifying targets of PROTACs
  • Analyse the physical principles that govern the use of light microscopy methods for the characterization of proteins in living cells.
  • Apply different microscopy methods commonly used for analysis of cellular protein signalling events, including the use of advanced imaging analysis and live cell imaging.



  • Identify and evaluate advantages and limitations of the use of above listed methods.
  • Justify the use of PROTACs and mass spectrometry in understanding the mechanisms of small molecule-based drugs or drug candidates
  • Understand the central aspects of translational tools and be able to discuss and communicate these to other scientists, clinicians, and the public

The course will be organized with a mixture of scientific seminars by invited speakers including lectures about stem cells, PROTACs, CRISPR-based genome engineering, high-resolution microscopy, cryo-EM, as well as the applications of modern proteomics technologies for drug discovery. In addition, the course will include group work, demonstrations, and practical exercises provided and supervised by leading specialists in the field.

The next generation of CRISPR-Cas technologies and applications. Pickar-Oliver A, Gersbach CA.Nat Rev Mol Cell Biol. 2019 Aug;20(8):490-507. PMID: 31147612

Chemoproteomics and Chemical Probes for Target Discovery.

Drewes G1, Knapp S2. Trends Biotechnol. 2018 Dec;36(12):1275-1286.

PMID: 30017093


Induced pluripotent stem cells in disease modelling and drug discovery.

Rowe RG, Daley GQ. Nat Rev Genet. 2019 Jul;20(7):377-388. PMID: 30737492


PROteolysis TArgeting Chimeras (PROTACs) - Past, present and future. Pettersson M1, Crews CM2. Drug Discov Today Technol. 2019 Apr;31:15-27. 13. PMID: 31200855

Participants must meet the admission criteria in BRIDGE - Translational Excellence Programme

Continuous feedback during the course of the semester
Type of assessment
Continuous assessment
Course participation
Attendance and active participation
All aids allowed
Marking scale
passed/not passed
Censorship form
No external censorship
Criteria for exam assessment


  • Understand the applications of stem cells and PROTACs in drug discovery
  • Understand the principles of gene editing



  • Interpret the experimental data obtained by the above-mentioned methods.
  • Present experimental data in an understandable and scientifically sound manner.
  • Discuss and critically review articles and research proposal for appropriateness in the utilization of methodological approaches for characterization of protein function in vivo.
  • Design a tailored research plan to test specific hypotheses related to the characterization of protein function in mammalian cells using relevant and informative methods



  • Project planning.
  • Data acquisition and interpretation.
  • Scientific communication both written and oral
  • Scientific communication both written and oral

Part time Master and Diploma courses

  • Category
  • Hours
  • Lectures
  • 10
  • Preparation
  • 5
  • Theory exercises
  • 5
  • Exercises
  • 20
  • English
  • 40