Postgraduate Specialisation in Clinical Pathology and Clinical Biochemistry

Molecular biology

Course code

4S00800

Name of lecturer

Donato Zipeto

Number of ECTS credits allocated

2

Other available courses

• Scuola di Specializzazione in Patologia clinica e Biochimica clinica (accesso riservato ai "non medici" - D.I. 716/2016)

Academic sector

BIO/11 - MOLECULAR BIOLOGY

Language of instruction

Italian

Site

VERONA

Period

not yet allocated

Learning outcomes

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 trials clinical trials based on gene therapy, disappointments and hopes, 7) current and future perspectives Upon completion of the course, students will: - have a basic knowledge of zoonoses and viruses - 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

• 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) • Herpes vectors and poxviruses • Retroviral and lentiviral vectors • Clinical trials: hematological, metabolic, neurodegenerative, neuromuscular and dystrophies, cystic fibrosis • Anti-tumor gene therapy, oncolytic viruses

Assessment methods and criteria