Advanced treatment for rare neurological “RASopathies” Parents’ initiative for affected children leads to a new EU research project RASopathies are a group of genetic diseases triggered by increased activation of the cellular RAS-MAPK signal pathway. This signal pathway has a decisive influence on growth and the differentiation of our cells.…..
Application of base editors in organoids opens new doors for cancer research The development of new cancer treatments depends on suitable tumor models in the laboratory. Researchers from the Organoid group (Hubrecht Institute) and the Princess Máxima Center have used base editors to simultaneously introduce multiple cancer-related mutations into healthy…..
Efficacy of gene therapy demonstrated in patients suffering from Crigler-Najjar Syndrome Crigler-Najjar syndrome is characterized by hyperbilirubinemia. If it is not treated quickly, the build-up of bilirubin, due to a deficiency in the liver enzyme UGT1A1, can cause significant neurological damage and become fatal. At the current time, the only…..