35799 - Gene Delivery and Bioinformatics

Learning outcomes

This course gives an overview of the field of human and animal gene therapy. It covers the fundamentals of using gene delivery for treating human and animal diseases, and details the viral vectors that are used for gene delivery, including their generation and use, and the methods that are used for controlling therapeutic gene expression. An overview on the possibility of using animals as model for  spontaneus pathologies will be given as well as details on the regulatory issues covering Advanced Therapy Medicinal Products (ATMP), the role of EMEA and the standard procedure sto carry our an environmental risk assessment (ERA).

Course contents

Introduction and Course Description

Definition

Gene Delivery

 

3. Objectives

 

4. Delivery of Therapeutic genes

 

5. Gene Therapy Targets

 

6. Delivery modes

 

7. Steps in Gene Therapy

 

8. Gene Therapy Viral Vectors

 

 

Viral Vectors

 

11. Objectives

 

Retroviral Life Cycle

Retroviral Genome Structure

Generation of Recombinant Retrovirus

Application of Retroviral Vectors

 

Adenovirus

Genome Structure

Adenovirus life cycle

Generation of recombinant adenovirus

Application of Adenoviral Therapy

 

Adeno-associated Virus

Genome Structure

life cycle

Generation of recombinant adeno-associted virus

Application of AAV Therapy

 

Herpesvirus

Genome Structure

life cycle

Generation of recombinant Herpesvirus

Application of Herpesviral Therapy

 

Poxvirus

Genome Structure

life cycle

Generation of recombinant poxvirus

Application of poxvirus Therapy

 

Comparison of Viral Vectors

 

REGULATORY ISSUES for ATMP

ERA

 

Readings/Bibliography

Suggested papers and  web site resources

Teaching methods

lectures and laboratory practicals

Assessment methods

oral test

Teaching tools

slides and scientific papers

Office hours

See the website of Alessandra Scagliarini