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

Introduction to gene therapy.

Sources of scientific information and documentation.

Introduction to gene transfer.

Transductional targeting; transcriptional targeting; inducible and tissue-/tumor-specific promoters.

Genome editing using CRISPR-Cas.

Laboratory 1: bioinformatics tools for CRISPR-Cas applications.

Viral vectors for gene therapy: adenoviruses, adeno-associated viruses, and retroviruses.

Non-viral methods for gene transfer.

Gene silencing strategies: antisense oligonucleotides, RNA interference, and ribozymes.

Therapeutic applications of non-coding RNAs.

Major applications of gene therapy for monogenic inherited diseases.

Gene and cell therapy for cancer.

Ethical and legal aspects of somatic, fetal, and germline gene therapy.

Laboratory 2: group work and classroom discussion on controversial aspects of gene therapy.

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.

If the student has taken part in the in-class activities, the grade obtained from these activities will contribute to the final grade.

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

See the website of Chiara Pirazzini