MOLECULAR THERAPY
cod. 1000221

Academic year 2024/25
1° year of course - Second semester
Professor
Giovanni ROTI
Academic discipline
Malattie del sangue (MED/15)
Field
Discipline medico-chirurgiche e riproduzione umana
Type of training activity
Characterising
42 hours
of face-to-face activities
6 credits
hub:
course unit
in ITALIAN

Learning objectives

Explain the basic concepts of molecular biology relevant to therapeutic strategies.
Describe the different types of molecular therapies and their mechanisms of action.
Small molecule
Antibody-based strategies
Epigenetics
CAR-T therapy
Tyrosine kinase
Discussing several disease models
Preclinical and clinical phases
HT screening
Principle of pharmacology
Principle of clinical trial
Protein-protein interaction and therapy
Hormone and therapy

Prerequisites

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Course unit content

This course explores the exciting field of molecular therapy, which utilizes molecules to diagnose, treat, and prevent diseases. We will delve into the fundamental principles behind various approaches, including gene therapy, RNA interference, small molecule therapeutics, antibody-based therapies, and cell and gene editing. The course will also address the ethical considerations, regulatory processes, and future directions of this rapidly evolving field.

Full programme

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Bibliography

PDF provided by lecturer

Teaching methods

Introduction to Molecular Therapy
The Central Dogma and Molecular Targets
Small Molecule Therapeutics
Drug Discovery & Development Process
Antibody-Based Therapies
Monoclonal Antibodies & Immunotherapy
Cell and Gene Editing Technologies
CRISPR-Cas9 & Applications in Therapy
Gene Therapy: Concepts & Vectors
Viral & Non-Viral Vectors for Gene Delivery
RNA Interference (RNAi) Technology
Applications of RNAi Therapeutics
Ethical Considerations & Regulatory Processes
The Future of Molecular Therapy
Final Exam

Assessment methods and criteria

Written and oral test

Other information

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2030 agenda goals for sustainable development

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