About GetRadi

The goal of the network “Gene Therapy of Rare Diseases” (GetRadi) is to train 10 PhD students to become exceptional experts in gene therapy of rare diseases.

A rare disease is a disease that affects less than 1 in 2000 people. However, since more than 6000 rare diseases are known, approximately 300 million people are suffering from rare diseases worldwide - including 30 million in the EU alone. Most of these rare diseases are caused by a genetic mutation and therefore a genetic therapy based on gene augmentation or, ideally, direct gene correction, is the only possibility to offer a permanent cure. However, only very few gene therapy products have reached the market up to now. While this is proof-of-principle for the potential of gene therapies, it also illustrates that there is an urgent need for the development of many more of them, especially at more affordable prices.

To achieve this, novel ideas are required to solve different technical problems associated with multifaceted nature of gene therapy, but also researchers with an excellent education in all technologies associated with gene therapies including rare disease models. Therefore, the overall goal of our network Gene Therapy of Rare Diseases (GetRadi) is to train 10 PhD students to become exceptional experts in gene therapy of rare diseases. In their research projects, the ESR will aim to overcome major technical challenges that still prevent the widespread use of somatic gene therapy in the clinic. All projects are embedded in a tight and synergistic academic-industrial collaboration to develop marketable products for the application of gene therapy. This GetRadi training will allow young scientists to spearhead future research efforts in gene therapy of rare diseases and thus increase the speed that desperately needed novel gene therapies are developed.

Funded by the European Union. Views and opinions expressed are however those of the author(s) only and do not necessarily reflect those of the European Union or the European Research Executive Agency. Neither the European Union nor the granting authority can be held responsible for them.