pChronic diseases based on individual gene mutations constitute a major burden for the individual and society. Advances in gene diagnostics have produced a wealth in knowledge for the molecular basis of disease. However, the translation of these insights into novel ATMPs and related therapeutics is lagging behind. In the past years, break-through innovations have been made in gene therapy employing viral vectors which will lift the general scepticism towards this type of therapy. Still, the hurdle for gene therapy is high as the intervention is irreversible. Therefore, in order to enable a broader and more rapid implementation of gene therapy, safe and powerful means are required to assess the potential benefits and risks of gene therapy. This project aims to prove the use of therapeutic mRNA as a safe and powerful therapeutic modality to assess the potential risk/benefit of viral gene therapy in the individual and/or the patient group. Messenger RNA has unique characteristics for this purpose: The therapeutic effect will be achieved through protein expression, thus in the same way as for viral gene therapy. However, in contrast to gene therapy, this change is reversible. Should the desired therapeutic effect not be achieved, or should dosing turn out to be critical or immunogenicity be observed, then the intervention can be stopped without persistent risk for the patient. Together with the Belgian project partners we have identified the congenital chronic disease cystinosis as a highly suited test case. Success in this indication paves the way to accelerated development and approval of novel medications for the more than 6,000 congenital diseases, which often are severely debilitating or fatal. | In dit project kan het bedrag onder Rijksbijdrage ook deels afkomstig zijn van andere subsidieverstrekkers. Hierbij valt te denken aan Europese subsidies zoals EFRO./p
