76211 - Gene Therapy

Learning outcomes

Define the main technologies of gene transfer and compare them in terms of therapeutic approaches, efficiency and safety. Explain inherited and acquired diseases that could benefit from gene therapy. Discuss the regulatory aspects related to gene therapy. Choose the gene therapy approach that fits each disease, and evaluate its advantages and disadvantages. Critically evaluate features, innovation, safety of reports on gene therapy studies. Keep up-to-date on rules, methods, technologies and devices, through scientific literature, gene therapy websites and data banks.

Course contents

Gene therapy fundamentals and sources

Fundamentals of gene transfer

Genome editing with CRISPR-Cas9

Gene therapy lab 1: informatic tools for CRISPR-Cas9 technology

Viral vectors for gene therapy

Non-viral gene transfer

Non-coding RNA therapeutics

Targeting: transductional targeting; transcriptional targeting; Inducible and tissue-specific promoters

Ablative strategies: Transduction of antisense constructs; Antisense oligonucleotides; Ribozymes; Intracellular antibodies; RNA interference; engineered nucleases.

Cancer gene therapy

Gene therapy of inherited of monogenic inherited diseases

Gene therapy of infectious diseases

Safety issues in preclinical and clinical gene therapy; Italian and international laws and safety; Ethic issues concerning fetal or germinal cell gene therapy

Gene therapy lab 2: work group on gene therapy clinical applications

Readings/Bibliography

Original research articles and provided by the lecturer as .pdf files.

Pdf files of lectures

Chapters from Strachan, Genetics and Genomics in Medicine, Garland Science

Teaching methods

The course will be structured as follows

-frontal lectures with PowerPoint slides

-class-room exercises on gene therapy applications

-discussion of original research articles on topics related to the lectures

Assessment methods

Student learning will be checked at the end of the course by an oral exam.

The final mark will be calculated as the weighted mean (based on CFUs) of the marks obtained in all the courses of TERAPIE MEDICHE INNOVATIVE (C.I.).

A mark above or equal to 18/30 is required to obtain the final assessment.

Teaching tools

Teaching slides and materials will be available on line at Unibo e-learning website https://iol.unibo.it [https://iol.unibo.it/] upon registration with Unibo institutional credentials.

Office hours

See the website of Manuela Ferracin