Moving the first steps towards the development of gene therapy for charcot-marie-tooth disease type 2a

The BNL Foundation, in the context of its initiative “A friendly hand to employees’ projects”, has recently funded the progress of scientific research about Charcot-Marie-Tooth disease type 2A (CMT2A).

In the past years, thanks to the support from the Association “ProgettoMitofusina 2”, the Neural Stem Cells Lab of “Centro Dino Ferrari”, University of Milan, directed by Professor Stefania Corti, has published significant results on renowned scientific journals, giving a valuable insight into the still obscure pathogenic basis of the disease. Alongside this project, the team of Professor Corti is now working on a possible therapeutic approach to CMT2A, an incurable disease so far. To this day, gene therapy represents a promising strategy, as it aims to replace the damaged gene that causes the disease with a healthy copy. This therapeutic strategy has already given encouraging results in other neuromuscular diseases. Therefore, the aim is to use the same approach in order to develop a successful treatment for CMT2A.

Thanks to the valuable grant received from BNL Foundation, the team had the opportunity to buy the essential equipment for this study, laying the foundations for a future application on patients.

More specifically, purchased equipment included: