The overall objective of the course is to make the student able to: 1) learn the fundamental concepts of gene therapy and gene replacement; 2) have an overview on RNA therapeutics with a specific focus on ASOs, siRNAs, SINEUPs, APTAMERS and deadCas9 technlogies; 3) know about drug-repositioning approaches. As learning outcomes, we expect to train a future biotechnologist able to effectively apply these approaches in the context of personalized medicine.
Course Prerequisites
Good knowledge in the field of general and molecular biology and human genetics: structure and function of DNA and RNA; protein synthesis; genes and chromosomes; patterns of inheritance (dominant/recessive/x-linked); genetic mutations and pathogenetic mechanisms associated with diseases.
Teaching Methods
Frontal lessons. The teaching material will be made available to the students (Kiro platform); the lessons will be organized in such a way that all the topics are supported by practical examples, demonstrating how the topic under discussion may have a practical application in their future professional activity. Teacher-students interaction will be stimulated as much as possible.
Assessment Methods
Written exam lasting 60 minutes, consisting of 4 open-ended questions covering all topics discussed during lectures. Evaluation scale: 0-31 (30 with honors). The exam is considered passed with a score of at least 18 out of 30. The result of the Gene Therapy exam will be published on Esse3, where students can accept or refuse the grade. For registration, students must enroll directly through their personal area. The final grade for the “NON PHARMACEUTICAL THERAPIES I” course will be recorded in the student’s curriculum and will represent the weighted arithmetic mean of the grades obtained for Gene Therapy (3 CFU), Immunotherapy (3 CFU), and Viruses and Vaccines (3 CFU) modules.
Texts
Title:RNA Therapeutics . The Evolving Landscape of RNA Therapeutics Authors:Giangrande - AAVV Editor:Elsevier - Edition:2022 Title: Handbook of Cell and Gene Therapy From Proof-of-Concept through Manufacturing to Commercialization Authors:Hazel Aranha, Humberto Vega-Mercado Editor:Routledge - CRC Press Edition:2023
Contents
The program will cover different topics in the field of gene therapy and personalized medicine: gene therapy by CRISPR/Cas9; gene replacement, RNA therapeutics (ASOs, siRNAs, epigenome editing by deadCas9, SINEUPs, miRNA agonists and antagonists, Aptamers), drug repositioning approaches; delivery strategies.
Course Language
English
More information
To make appointment, please contact: elisa.giorgio@unipv.it
