Since my medical school years I wanted to be a clinician scientist and during that time I was incredibly fascinated by stem cell research, gene therapy and their future clinical potential. San Raffaele was without a doubt at the forefront of those areas and I wanted to get a PhD there at all costs (indeed I was brave and possibly naive enough not to apply to any other institutions!). Luckily I then got it!
I obtained my PhD in 2010 in the laboratory of Professor Giulio Cossu at San Raffaele Scientific Institute. During my PhD I worked on human artificial chromosomes for the therapy of Duchenne muscular dystrophy. During that time I also started to work on another research project that was then further developed during my postdoctoral period, based upon pluripotent stem cell-derived myogenesis Our work was the first to show safe and efficacious pre-clinical gene replacement therapy with a human artificial chromosome into an animal model of Duchenne muscular dystrophy. We were also among the first to report on the therapeutic potential of disease-specific (i.e. limb-girdle muscular dystrophy 2D) iPS cells that were genetically corrected, transplanted back into an ad hoc generated mouse model and shown to provide therapeutic potential. I am currently working towards the clinical translation of these strategies into future therapeutic tools for muscle disorders.
The research environment during my PhD years was great. There was a high concentration of top-notch scientists within the same building (and frequently on the same floors!). There were also lots of opportunities for networking with fellow PhD candidates and this has certainly worked very well, as I met some of my current best friends during that time in the institute.
My PhD has certainly shaped my career significantly, as it provided me with excellent training and expertise, allowing to develop my current research programs and to become an independent investigator.