Biotechnology in Pharmacology
A.Y. 2025/2026
Learning objectives
The course is aimed at providing the student with a view of the biotechnological drugs already available for the treatment of various disease, and to provide the basis for the identification of new pharmacological targets and the design of novel biotechnological drugs.
Expected learning outcomes
At the end of the course the student will have a deep knowledge on the biotechnological drugs on the market, and she/he will be able to apply the knowledge to the identification of new targets and the design end evaluation of novel drugs.
Lesson period: Second semester
Assessment methods: Esame
Assessment result: voto verbalizzato in trentesimi
Single course
This course cannot be attended as a single course. Please check our list of single courses to find the ones available for enrolment.
Course syllabus and organization
Single session
Responsible
Lesson period
Second semester
Course syllabus
Experimental biology teaching unit (2 CFU)
1. Animal Cell Cultures. The culture of animal cells. How to culture cells from a tissue. The evolution of a culture: primary cultures, cell lines. Culture medium. Adherent cultures and suspension cultures.
2. Methods for Gene Transfer. Genetic manipulation of animal cells: principles and purposes. Transient and stable transfection. Methods to introduce genetic information into animal cells.
3. Applications of Cell Biology. Vectors for constitutive or regulated expression in mammalian cells. Reporter genes and selection markers. Studying gene function. Protein production. Fusion proteins. Studying localization. Studying the effects of mutations.
4. Methods for Gene Silencing. Gene silencing and knockout. RNA interference: mechanisms and applications. Small non-coding RNAs that regulate gene expression.
5. Genome Editing. Zinc Finger Nucleases (ZFNs). Transcription Activator-Like Effector Nucleases (TALENs). CRISPR/Cas9 System.
6. Stem Cell Biology. Definition of stem cells. Embryonic, fetal, and tissue (somatic or adult) stem cells. Potency and plasticity of stem cells. Adult stem cells. Induced pluripotent stem cells: generation, characterization, and differentiation. 3D cultures - organoids.
7. In vitro High Throughput Screening. HTS methods to identify biologically active molecules. Cell-based screenings. Examples of drug repurposing based on cell models.
8. Therapeutic Use of Cells. Applications of stem cells: regenerative medicine and in vitro tissue production. CAR-T cells for cancer therapies.
Pharmacology teaching unit (6CFU)
1. Nucleic acid drugs
a. Biological activities and methods of study of natural RNAs of pharmacological interest: mRNA, miRNA, lncRNA, circRNA
b. Biological activities and methods of study of synthetic nucleotide polymers: aODN, siRNA, aptamers
c. Generation and methods of study of mRNA drugs: vaccines and replacement therapies
d. Pharmacokinetics, toxicology, functionalization and engineering
2. Recombinant proteins
a. Recombinant proteins and fusion proteins
b. Monoclonal antibodies and antibody derivatives: biology, production, engineering
c. Pharmacokinetics, toxicology, functionalization and engineering
3. Pharmacological biotechnology applied to the clinic
a. Oncological therapy
b. Immunosuppressive therapy
c. Immunostimulatory therapy
4. Biotechnology in the study of gene transcription drugs
a. Intracellular receptors
b. The study of protein-protein interactions and protein-DNA binding
c. Generation of cellular models
d. Generation of animal models
5. Biotechnology in preclinical research
a. Successful examples of biotechnological research in pharmacology with virtual exercises
6. Practical exercises in biotechnology in pharmacology
a. Transfection of cells grown in culture and analysis of the activity of reporter genes regulated by ligand-operated transcription factors following dose-response assays
1. Animal Cell Cultures. The culture of animal cells. How to culture cells from a tissue. The evolution of a culture: primary cultures, cell lines. Culture medium. Adherent cultures and suspension cultures.
2. Methods for Gene Transfer. Genetic manipulation of animal cells: principles and purposes. Transient and stable transfection. Methods to introduce genetic information into animal cells.
3. Applications of Cell Biology. Vectors for constitutive or regulated expression in mammalian cells. Reporter genes and selection markers. Studying gene function. Protein production. Fusion proteins. Studying localization. Studying the effects of mutations.
4. Methods for Gene Silencing. Gene silencing and knockout. RNA interference: mechanisms and applications. Small non-coding RNAs that regulate gene expression.
5. Genome Editing. Zinc Finger Nucleases (ZFNs). Transcription Activator-Like Effector Nucleases (TALENs). CRISPR/Cas9 System.
6. Stem Cell Biology. Definition of stem cells. Embryonic, fetal, and tissue (somatic or adult) stem cells. Potency and plasticity of stem cells. Adult stem cells. Induced pluripotent stem cells: generation, characterization, and differentiation. 3D cultures - organoids.
7. In vitro High Throughput Screening. HTS methods to identify biologically active molecules. Cell-based screenings. Examples of drug repurposing based on cell models.
8. Therapeutic Use of Cells. Applications of stem cells: regenerative medicine and in vitro tissue production. CAR-T cells for cancer therapies.
Pharmacology teaching unit (6CFU)
1. Nucleic acid drugs
a. Biological activities and methods of study of natural RNAs of pharmacological interest: mRNA, miRNA, lncRNA, circRNA
b. Biological activities and methods of study of synthetic nucleotide polymers: aODN, siRNA, aptamers
c. Generation and methods of study of mRNA drugs: vaccines and replacement therapies
d. Pharmacokinetics, toxicology, functionalization and engineering
2. Recombinant proteins
a. Recombinant proteins and fusion proteins
b. Monoclonal antibodies and antibody derivatives: biology, production, engineering
c. Pharmacokinetics, toxicology, functionalization and engineering
3. Pharmacological biotechnology applied to the clinic
a. Oncological therapy
b. Immunosuppressive therapy
c. Immunostimulatory therapy
4. Biotechnology in the study of gene transcription drugs
a. Intracellular receptors
b. The study of protein-protein interactions and protein-DNA binding
c. Generation of cellular models
d. Generation of animal models
5. Biotechnology in preclinical research
a. Successful examples of biotechnological research in pharmacology with virtual exercises
6. Practical exercises in biotechnology in pharmacology
a. Transfection of cells grown in culture and analysis of the activity of reporter genes regulated by ligand-operated transcription factors following dose-response assays
Prerequisites for admission
Knowledge of pharmacokinetics, pharmacodynamics and pharmacology of the main classes of drugs used in the clinic
Teaching methods
Classroom-taught lessons and practical exercises
Teaching Resources
Slides, experimental protocols and all teaching material are available through the MyAriel website of the course.
Books:
· FARMACI BIOTECNOLOGICI. Aspetti farmacologici e clinici dei medicinali biotecnologici. Vegeto E, Maggi A, Minghetti P. CEA Editore
· FARMACOLOGIA GENERALE E MOLECOLARE. Clementi E, Fumagalli G. Edra Editore
· CULTURE OF ANIMAL CELLS: A MANUAL OF BASIC TECHNIQUE AND SPECIALIZED APPLICATIONS. Ian Freshney R. John wiley & sons, inc.
Books:
· FARMACI BIOTECNOLOGICI. Aspetti farmacologici e clinici dei medicinali biotecnologici. Vegeto E, Maggi A, Minghetti P. CEA Editore
· FARMACOLOGIA GENERALE E MOLECOLARE. Clementi E, Fumagalli G. Edra Editore
· CULTURE OF ANIMAL CELLS: A MANUAL OF BASIC TECHNIQUE AND SPECIALIZED APPLICATIONS. Ian Freshney R. John wiley & sons, inc.
Assessment methods and Criteria
The experimental biology teaching unit exam consists of a written test comprising 10 multiple-choice questions (2 points for each correct answer and 0 points for each incorrect or unanswered question) and 1 open-ended question (up to 10 points). The test aims to: i) assess the level of knowledge and understanding of the topics covered in the lectures; ii) verify the ability to describe the topics clearly and, where required, using specific terms of the discipline.
The pharmacology teaching unit exam consists of two tests: a written test with 5 questions related with the topics covered in class (maximum points 20/30), and a scientific report of the protocols used and the results obtained in the practical exercises (maximum points 10/30). Both tests are aimed at verifying the objectives achievement, in terms of acquisition of knowledge and understanding of the subject, critical reasoning and narrative ability with appropriate vocabulary and metrics.
The final mark will consist of the weighted average, based on CFU, of the partial marks for the two didactic units.
The pharmacology teaching unit exam consists of two tests: a written test with 5 questions related with the topics covered in class (maximum points 20/30), and a scientific report of the protocols used and the results obtained in the practical exercises (maximum points 10/30). Both tests are aimed at verifying the objectives achievement, in terms of acquisition of knowledge and understanding of the subject, critical reasoning and narrative ability with appropriate vocabulary and metrics.
The final mark will consist of the weighted average, based on CFU, of the partial marks for the two didactic units.
BIO/13 - EXPERIMENTAL BIOLOGY - University credits: 2
BIO/14 - PHARMACOLOGY - University credits: 6
BIO/14 - PHARMACOLOGY - University credits: 6
Single bench laboratory practical: 32 hours
Lessons: 48 hours
Lessons: 48 hours
Professor(s)