AMC articles in MAGMA and Janus
In 2015 we presented our plans to investigate if gene therapy could be a feasible option for treating Crigler-Najjar syndrome, inherited jaundice, in MAGMA. To prevent brain damage patients suffering from this severe inherited liver disorder need whole body exposure to intense blue light each day for many hours, up to 14 hours. In this issue of MAGMA we report the results of the developed gene therapy strategy as presented in the recent NEJM paper. In this we show that the treatment is safe and effective and that patients that received the high vector dose could stop phototherapy for over a year now. In follow-up studies also younger patients will be treated using the higher dose. Furthermore, methods to prevent and/or overcome the hurdles imposed by the immune system will be investigated, to make gene therapy available to all CN patients including those having antibodies that block liver entry by the vector, about 30% of all CN patients.