Postgraduate Specialisation in Clinical Pathology and Clinical Biochemistry

Molecular biology

Course code

4S00800

Name of lecturer

Donato Zipeto

Number of ECTS credits allocated

2

Academic sector

BIO/11 - MOLECULAR BIOLOGY

Language of instruction

Italian

Site

VERONA

Period

not yet allocated

Learning outcomes

Provide the student with information on 1) principles and limits of gene therapy, 2) approaches and types of vectors, 3) notes on non-viral vectors, 4) gene editing (CRISPR/Cas9), 5) main viral vectors, advantages and disadvantages, 6) examples of clinical trials based on gene therapy, disappointments and hopes, 7) current and future perspectives. Upon completion of the course, students will have acquired the knowledge necessary to understand how viruses can be engineered and used as therapeutic agents (vectors for gene therapy , oncolytic viruses).

Syllabus

Gene therapy: • Introduction to gene therapy • Non-viral vectors, siRNA and miRNA, peptide vectors • Gene editing: ZFN, TALEN, Crispr/Cas9 • Viral vectors, general information • Adenovirus (AV) • Adeno-associated virus (AAV) • Vectors herpes and poxviruses • Retroviral and lentiviral vectors • Clinical trials: hematological, metabolic, neurodegenerative, neuromuscular and dystrophies diseases, cystic fibrosis • Anti-tumor gene therapy, oncolytic viruses

Assessment methods and criteria