GEne Therapy-VALidation of Individualized Dose, Auto-immunity and Therapeutic effect wIth OligoNucleotides. (GET VALIDATION)

This model 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.