Nanoparticles and Viral Vectors
A.Y. 2024/2025
Learning objectives
The course provides the student with knowledge about the most innovative strategies to design and develop biotechnological drugs for the cure of congenital or acquired pathologies.
Specific knowledge will be provided on usage and production of viral vectors for gene therapy and vaccines; nanoparticle design, functionalization, and characterization techniques; concepts and examples of biosensor applications.
Specific knowledge will be provided on usage and production of viral vectors for gene therapy and vaccines; nanoparticle design, functionalization, and characterization techniques; concepts and examples of biosensor applications.
Expected learning outcomes
At the end of the course the student should be able to:
· describe the characteristics of the different viral vectors discussed during the training;
· evaluate the type of viral vector most suitable for a specific class of pathologies;
· analyze the advantages and disadvantages of the different viral vectors discussed during class;
· compare the different types of inorganic and organic nanoparticles based on their physical and chemical properties and as function of the scale;
· specify nanoparticle characterization techniques and evaluate their limitations and scopes;
· describe the functioning principles of the main biosensor techniques based on nanoparticles;
· describe the design of nanoparticles for the delivery of biotechnological active ingredients;
· describe the methods and techniques of nanoparticle decoration;
· analyze the synthesis and functionalization methods of nanoparticles.
· describe the characteristics of the different viral vectors discussed during the training;
· evaluate the type of viral vector most suitable for a specific class of pathologies;
· analyze the advantages and disadvantages of the different viral vectors discussed during class;
· compare the different types of inorganic and organic nanoparticles based on their physical and chemical properties and as function of the scale;
· specify nanoparticle characterization techniques and evaluate their limitations and scopes;
· describe the functioning principles of the main biosensor techniques based on nanoparticles;
· describe the design of nanoparticles for the delivery of biotechnological active ingredients;
· describe the methods and techniques of nanoparticle decoration;
· analyze the synthesis and functionalization methods of nanoparticles.
Lesson period: Second semester
Assessment methods: Esame
Assessment result: voto verbalizzato in trentesimi
Single course
This course can be attended as a single course.
Course syllabus and organization
Single session
Responsible
Lesson period
Second semester
Course syllabus
1_Prof. Lara Manganaro
Didactic Unit: Viral Vectors
This unit provides an in-depth exploration of viral gene transfer techniques and gene therapy. The main themes include:
Viral Replication and Gene Expression Strategies: Comprehensive study of in vivo and ex vivo gene therapy methodologies.
Characteristics and Applications of Vectors: Detailed examination of retroviral, lentiviral, adenoviral, and adeno-associated virus (AAV) vectors, highlighting their unique characteristics and uses.
Innate Immune Response: Analysis of the body's innate immune response to viral vectors.
Advanced Applications: Coverage of viral vector use in genetic editing, genetic vaccines, and oncolytic viruses.
2_Prof. Tommaso Pietro Fraccia
2 Didactic Unit: Physics
Inorganic nanoparticles (metallic, magnetic, semiconductors, quantum dots), physical properties as a function of scale, diffusion, and their use for imaging. Hyperthermia.
Physical principles for the description of the collective phase behavior of polymeric systems (phase separations, complex coacervation), self-assembly of molecules and biological macromolecules (lipids, nucleic acids, and peptides), and nanostructured biomaterials (DNA nanotechnology). Application examples for the development of organic nanoparticles for the transport and/or controlled release of drugs: liposomes, polymeric nanoparticles, polymeric micelles, polymersomes, dendrimers, polymer/lipid-nucleic acid complexes.
Techniques for the characterization of nanoparticles: electron microscopy (TEM, SEM), atomic force microscopy (AFM), dynamic light scattering (DLS), Z-potential, radiation scattering (neutrons, X-rays). Introduction to microfluidics and examples of lab-on-chip applications.
Nanoparticle-based biosensors (SPR, enhanced Raman scattering, fluorescence, light scattering).
3_Prof. Sergio Romeo
3 Didactic Unit: Chemistry of Nanoparticles
Introduction: properties of nanoparticles and pharmacokinetics, applications of nanoparticles in gene- and drug-delivery.
Inorganic particles: calcium sulfate, silica, gold, magnetic compounds, quantum dots. Carbon nanotubes, fullerenes, supramolecular systems.
Synthetic or natural biodegradable particles: cationic lipids, lipid nano emulsions, solid lipid nanoparticles, peptide-based nanoparticles.
Polymeric vectors: cationic polymers, natural proteins, peptides, polysaccharides, dendrimers, polyethylenimine (PEI).
Active and passive targeting: examples of site-specific release strategies, design of bioconjugates; vascular targeting; enzyme-mediated tissue selectivity; enzyme-prodrug therapies, central nervous system targeting.
Didactic Unit: Viral Vectors
This unit provides an in-depth exploration of viral gene transfer techniques and gene therapy. The main themes include:
Viral Replication and Gene Expression Strategies: Comprehensive study of in vivo and ex vivo gene therapy methodologies.
Characteristics and Applications of Vectors: Detailed examination of retroviral, lentiviral, adenoviral, and adeno-associated virus (AAV) vectors, highlighting their unique characteristics and uses.
Innate Immune Response: Analysis of the body's innate immune response to viral vectors.
Advanced Applications: Coverage of viral vector use in genetic editing, genetic vaccines, and oncolytic viruses.
2_Prof. Tommaso Pietro Fraccia
2 Didactic Unit: Physics
Inorganic nanoparticles (metallic, magnetic, semiconductors, quantum dots), physical properties as a function of scale, diffusion, and their use for imaging. Hyperthermia.
Physical principles for the description of the collective phase behavior of polymeric systems (phase separations, complex coacervation), self-assembly of molecules and biological macromolecules (lipids, nucleic acids, and peptides), and nanostructured biomaterials (DNA nanotechnology). Application examples for the development of organic nanoparticles for the transport and/or controlled release of drugs: liposomes, polymeric nanoparticles, polymeric micelles, polymersomes, dendrimers, polymer/lipid-nucleic acid complexes.
Techniques for the characterization of nanoparticles: electron microscopy (TEM, SEM), atomic force microscopy (AFM), dynamic light scattering (DLS), Z-potential, radiation scattering (neutrons, X-rays). Introduction to microfluidics and examples of lab-on-chip applications.
Nanoparticle-based biosensors (SPR, enhanced Raman scattering, fluorescence, light scattering).
3_Prof. Sergio Romeo
3 Didactic Unit: Chemistry of Nanoparticles
Introduction: properties of nanoparticles and pharmacokinetics, applications of nanoparticles in gene- and drug-delivery.
Inorganic particles: calcium sulfate, silica, gold, magnetic compounds, quantum dots. Carbon nanotubes, fullerenes, supramolecular systems.
Synthetic or natural biodegradable particles: cationic lipids, lipid nano emulsions, solid lipid nanoparticles, peptide-based nanoparticles.
Polymeric vectors: cationic polymers, natural proteins, peptides, polysaccharides, dendrimers, polyethylenimine (PEI).
Active and passive targeting: examples of site-specific release strategies, design of bioconjugates; vascular targeting; enzyme-mediated tissue selectivity; enzyme-prodrug therapies, central nervous system targeting.
Prerequisites for admission
Basic notions of general biology, medicinal chemistry, organic chemistry applied to macromolecules and physics are required
Teaching methods
Lectures
Teaching Resources
Giacca M. "Gene Therapy"
Springer, 2010
ISBN 978-88-470-1643-9
Nanocarriers for drug delivery: concepts and applications
Springer, 2021
ISBN 3-030-63389-6, available online https://www.sba.unimi.it/
Slides of the course uploaded on Teams website
Springer, 2010
ISBN 978-88-470-1643-9
Nanocarriers for drug delivery: concepts and applications
Springer, 2021
ISBN 3-030-63389-6, available online https://www.sba.unimi.it/
Slides of the course uploaded on Teams website
Assessment methods and Criteria
The exam consists of 3 tests, one for each instructional unit. Viral Vectors Unit (3 CFU): written "multiple choice" test with one open-ended question. Physics Unit (2 CFU): oral exam. Chemistry Unit (2 CFU): oral exam.
The student should be able to present the acquired knowledge, as well as the ability to reason and integrate the topics covered in the lessons.
The final grade (out of thirty) will be calculated as the weighted average (based on the CFU) of the scores obtained in the individual instructional units.
The student should be able to present the acquired knowledge, as well as the ability to reason and integrate the topics covered in the lessons.
The final grade (out of thirty) will be calculated as the weighted average (based on the CFU) of the scores obtained in the individual instructional units.
BIO/19 - MICROBIOLOGY - University credits: 3
CHIM/08 - PHARMACEUTICAL CHEMISTRY - University credits: 2
FIS/07 - APPLIED PHYSICS - University credits: 2
CHIM/08 - PHARMACEUTICAL CHEMISTRY - University credits: 2
FIS/07 - APPLIED PHYSICS - University credits: 2
Lessons: 56 hours
Professor(s)
Reception:
Monday 10:30-12:30, by appointment via email
Via Mangiagalli 25, second floor, office 2062