Developing novel strategies for the prevention and treatment of disease

Molecular Therapy

The mission of TIGEM’s Molecular Therapy (MT) research plan is to develop novel strategies for the prevention and treatment of disease. One of our major research strategies is in vivo vector-mediated gene therapy. The general objective is to develop new vector systems using adeno-associated vectors (AAV) and helper dependent adenoviral vector (HDad) delivery methods, test them in the appropriate animal models, and translate the most promising approaches to the clinic.

Here at TIGEM, the AAV vector system has been particularly successful when used in several pre-clinical studies on lysosomal storage disorders (LSDs) and in a vast series of animal models of retinal degenerations. We also utilize small molecules such as specific chaperones for the treatment of LSDs.

Coordinator: Alberto Auricchio

AAV Gene Therapy
Alberto Auricchio

Our goal is to develop novel in vivo gene therapy and genome editing strategies for a wide range of inherited blindness and metabolic diseases.


Inborn Errors of Metabolism
Nicola Brunetti

As a paediatrician and geneticist, the overall goals of my research are to understand how defects in biochemical pathways result in disease and to develop novel therapeutic approaches for inborn errors of metabolism.

Phone +3908119230661

Behavioral Core
Elvira De Leonibus

My group aims to identify early behavioural and disease mechanisms in animal models of complex human disorders, with the ultimate goal of discovering and testing novel therapeutic strategies to manage them.


Mitochondrial Medicine
Alessia Indrieri

The overall goal of my research is to find effective treatments for defective mitochondrial functions. In particular we aim to develop mutation-independent strategies to treat rare and common mitochondrial-associated disorders.


Innovative Therapies for Lysosomal Diseases
Giancarlo Parenti

Interacting with patients gives me strong and enduring motivation to perform research at TIGEM, with the aim to identify novel therapeutic opportunities and alleviate the impact of diseases on patients’ lives.


Generation of Animal Models Facility
Nunzia Pastore

My research focuses on dissecting the molecular pathways underlying the pathology of liver disease associated with genetic disorders by combining 3D models and in vivo studies. TIGEM offers unique expertise and excellent resources to pursue my goal.

Phone+39 08119230630

Liver-Directed Gene Therapy
Pasquale Piccolo

Understanding and treating rare genetic diseases is a wonderful journey and an extraordinary creative adventure that teaches us a lot about human life and health.


Advanced Histopathology Facility
Nicolina Cristina Sorrentino

My research goal is to understand the mechanism of the neuropathology progression and develop advanced gene therapy and drug therapy protocols for the treatment of inherited neurodegenerative disorders

Phone+39 08119230649

Gene therapy for retinal diseases
Ivana Trapani

Having the possibility to help people, by finding solutions to diseases which are still untreatable, motivates me incredibly and makes me feel that my efforts are focused in a positive direction.