Targeted engineering of the human genome opens up an enormous number of new research opportunities and therapeutic possibilities. We are just at the beginning of the “age of genome editing” and we have already witnessed the amazing potential of these technologies to transform research, making possible to design and execute an unprecedented arrays of experimental studies, and attract from their very first steps amazing interest and resources from the biotechnological and pharmaceutical sectors as well as from less risk-adverse venture capital.
We thus expect that a fallout of the technological advances brought forward by UPGRADE will be to foster new fundamental research interrogating gene function and regulation also in previously intractable cells and tissues, and to trigger new investments in the biotechnological space aimed at capturing and exploiting their novel R&D potential.
Even more importantly, UPGRADE will provide proof-of-principle of several new therapeutic strategies developed and validated in paradigmatic disease models that could be expanded upon suitable adaptation to the treatment of many more disease entities. It should be noted that despite the promise of gene and cell therapy to become a new pillar of medicine in the near future, there are still few target tissues and disease settings that provide for feasible and effective exploitation of these powerful modalities of biological and genetic manipulation. UPGRADE will launch its investigation starting right from where the promise of gene and cell therapy has already brought some fruits, with the aim of overcoming the hurdles that currently limit the harvest only to the lowest hanging fruits.