Advanced Human Therapies - MEB

T - Theoretical; TP - Theory and practice; PL - Laboratory; S - Seminar; OT - Tutorial.

• Learning outcomes of the curricular unit:

  knowledge of Bio-pharmaceutical substances as innovative therapeutic way in present clinical practice.

  Adquire the basis of human gene therapy, including vectors and systems used for the applied examples, overview its limitations and advantages over conventional therapies.

  Review the basis for pharmacogenomics personalized therapy as well as the advantages and limitations associated with this technology.

  Raising awareness of bioethical issues associated with genetic testing, gene therapy, pharmacogenomics and protection of intellectual property in biotechnology products and processes.

• Syllabus:

  1. Concepts of general pharmacology and strategies for new drugs development.

  2 Biopharmaceutical substances: Main characteristics. Protein engineering andpost-translational modifications. Biosimilar. PAT and QbD.

  3 Gene Therapy: Vectors-enhanced Systems; Security; Production of gene therapy. Gene silencing: RNA interference technology (RNAi). Gene therapies associated with cell & tissue engineering.

  4 Pharmacogenomics: Genetic mechanisms. Examples of the variation in the patient sensitivity to drugs due to genetic polymorphisms. Application examples in personalized therapy, for biomarkers and development of new drugs.

  5 Bioethics associated with genetic testing, gene therapy, pharmacogenomics and protection of intellectual property in biotechnology products and processes.

• Demonstration of the syllabus coherence with the curricular unit's objectives

  In order to students understand the Advanced Therapies, in this unit general concepts of pharmacology and process development of new drugs will be discussed. Then,  protein engineering using nucleic acids and proteins will be addressed as an innovative application. Its limitations and advantages will be also addressed.

  Afterwards notions of gene therapy by Key-vectors, its security issues will be reviewed as well as some application examples that already are in clinical trials. After that, the gene therapy,  pharmacogenomics concepts for personalized human therapy will be presented as well its advantages and limitations. Along that, the ethical issues in the application of genetic testing and gene therapy, pharmacogenomics and intellectual property protection for biotechnology products and processes will  be discussed.

• Teaching methodologies (including evaluation):

  Teaching methodologies:

  In this curricular unit two types of class were used: expositive to explore the theoretical concepts, and active lessons  (TP or Lab) involving the participation of students in problem solving in order to explore aspects of each principal physiological system.

   

  The evaluation is based on a continuous evaluation (CE) and by an Exam (WE), as follows:

   

  Continuous evaluation (CE):

  The continuous evaluation is constituted by the accomplishment of an oral presentation (OP) of an scientific article concerning an technique used to study a physiological system function, and by Laboratory reports (LAB),  as follows:

   

  CE = 0.5OP + 0.5LAB

  The written exam (WE) is  2h long, and to be approved, Students must have a score WE >=9.5

   

  Formula for the calculation of Final Score (FS) is:

  FS = 0,3 CE + 0,7 WE. To be approved, Students must have a FS >= 9,5

  Rounded to units. By defect, beneath five tenths, per excess, from five tenths.

• Demonstration of the coherence between the teaching methodologies and the learning outcomes.

  The use of expository lectures and active classes in the learning process allows to understand  the application of theoretical concepts taught to students. During classes students are stimulated to participate in problem solving. In the problem solving  they connect the topics taught in this course and the topics taught in previous courses.

  The introduction of the continue component evaluation force students to maintain an ongoing study and monitoring during the semester contributing to the improvement of learning outcomes. The evaluation component exam involves all concepts transmitted allowing a correct evaluation of students that met the learning objectives stipulated.

• Main Bibliography

  Gabrielsson, J., Hjorth, S . Quantitative Pharmacology: An Introduction to Integrative Pharmacokinetic-Pharmacodynamic Analysis. Swedish Pharmaceutical Press, 2012.

  Khan, F.A. Biotechnology in Medical Sciences. CRC Press, 2014.

  Park, S.J. and Cochran, J.R. Protein Engineering and Design. CRC press. 2009.

  Templeton, N.S. Gene and Cell Therapy: Therapeutic Mechanisms and Strategies.  Third Edition. CRC Press, 2008.