In Trieste, the RNA Biology Group (M. Baralle) has a long-standing interest in the elucidation of the molecular mechanisms that control
An interesting collaborative study between the Mouse Molecular Genetics and Molecular Medicine groups in Trieste demonstrated how long-lasting gene therapy can be achieved. This was done by repeated gene transfer using serotype switching of the gene delivering Adeno Associated Virus and thereby attaining long-lasting therapeutic levels of expression of the gene product (Bockor et al., 2017, Gene Ther. 24, 649). This has very important consequences for treatment of the specific disease in question, Crigler-Najjar Syndrome Type 1, but also for gene therapy approaches in general.