Learning objectives
The course provides the student with the basics to understand the issues involved in the development of nucleic acids-based therapeutics and their potential in the application to different fields, from selective gene expression modulation to gene replacement therapy and vaccines production. Specific issues related to the development of these drugs will also be illustrated. At the end of the course the student will be able to classify and describe the different types and mechanisms of action of the main DNA and RNA biopharmaceuticals (KNOWLEDGE AND UNDERSTANDING) and explain the contents of the course systematically and with a language that makes use of the appropriate scientific terminology (COMMUNICATION SKILLS AND LEARNING SKILLS). Furthermore, the student is expected to have acquired the ability to integrate knowledge at the interface between different topic areas and developed critical awareness to manage the complexity of cutting-edge issues (ABILITY TO APPLY KNOWLEDGE AND UNDERSTANDING).
Prerequisites
Basic knowledge of biochemistry and biology.
Course unit content
The course will focus on the development of DNA and RNA-based therapeutic agents and their applications in gene therapy and as vaccines, with the aim of highlighting the topicality and potential use of this innovative class of biopharmaceuticals.
Full programme
• Nucleic acids: introduction and general aspects.
• General concepts on the different classes of DNA- and RNA-based therapeutic agents: antisense oligonucleotides (ASOs), small interfering RNAs and microRNAs (RNA interference), ribozymes, CRISPR-Cas9, aptamers, RNA decoys, SMaRT, messenger RNA, DNA and mRNA vaccines.
• Chemical modifications of nucleic acids.
• Targeting and delivery of nucleic acid-based biopharmaceuticals. Chemical conjugation and formulation using carriers. Non-viral vectors: different types of synthetic nanoparticles. Viral vectors and exosomes.
• Examples of therapeutic applications of the different classes of DNA and RNA biopharmaceuticals. Therapeutic agents for gene silencing or translation inhibition. Therapeutic agents that promote proteins production or antigen presentation. Special focus on vaccine against SARS-CoV-2.
Bibliography
The teaching material will be loaded on the Elly platform. The teaching material includes a copy of the slides and the webliography together with selected scientific articles.
Optional reference books:
1. RNA Therapeutics, The Evolving Landscape of RNA Therapeutics. Editors: P. Giangrande, V. de Franciscis, J. Rossi
2. Advances in Nucleic Acid Therapeutics. Editors: S. Agrawal, M. J. Gait
3. Molecular Biology of the Cell. B. Alberts.
Teaching methods
The teaching activities will be carried out in the form of in-person lectures. The teaching activities will be carried out through lectures with the help of slides that will be made available to the students on the Elly website.
Assessment methods and criteria
The comprehension of the covered topics will be assessed by a short presentation of a case study agreed upon with the teacher, either in group or individually.
Other information
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