90337 - Gene therapy

Learning outcomes

Know the basic concepts of gene therapy and genome editing. Understand the main gene transfer technologies, therapeutic strategies, efficiency and safety issues. Describe inherited and acquired diseases that could benefit from gene therapy. Identify potential targets and approaches for gene therapy; be able to critically evaluate features, innovation, safety in gene therapy studies. Learn how to present a gene therapy approach, from the pathology description to conventional and biotechnological strategies. Discuss the ethical issues related to gene therapy and genome editing. Critically evaluate research articles pertinent to gene therapy.

Course contents

Gene therapy fundamentals and sources

Fundamentals of gene transfer

Viral vectors for gene therapy

Non-viral gene transfer

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.

Genome editing with CRISPR-Cas9

Cancer gene therapy

Gene therapy of inherited of monogenic inherited diseases

Gene therapy of infectious diseases

Gene therapy of ocular diseases

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

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