The project Unravelling combined RNA interference and gene therapy in vitro and in vivo disease models as a potential Therapeutic strategy for CMT2A by DR Federica Rizzo has won a funding of 449.000 Euro in the last “call for aimed research” of the Italian Ministry of Health. 

The gene therapy project for CMT2A disease (Charcot-Marie-Tooth type 2A), followed by the Neural Stem Cell Laboratory of the Dino Ferrari Center under the supervision of Prof. Stefania Corti, was chosen together with other 53 projects within the category “theory enhancing” (biomedical translational research) dedicated to young researchers.

A total of 1,719 projects were submitted by researchers working in National Health Service structures, all of which peer reviewed by international auditors and, then, submitted to specific scientific panels for further verification and for validation of the merit ranking.

The goal of the gene therapy showed in this project for CMT2A disease is to “switch off” the defective Mitofusine gene and allow the expression of the healthy gene. In this way, patients should have a proper level of the protein so their cells could operate efficiently.

The large sum, along with the economic and emotional support of both Mitofusin 2 Project Association and Dino Ferrari Center Association, is an additional incentive to try harder and find a definitive gene therapy for patients with CMT2A disease.