NederlandsTopic
#Gentherapie
-
Researchers wake up silent, healthy genes in Prader-Willi syndrome
In Prader-Willi syndrome a group of genes does not work, even though every cell has a healthy back-up copy. Researchers in Melbourne switched that copy back on by removing one protein, in patient cells and in mice. Work on a medicine is now under way.
-
Precise DNA correction used inside a patient's body for the first time
Prime Medicine has for the first time treated a patient with prime editing directly inside the body. A single infusion is meant to repair a DNA error in the liver in Wilson's disease, an inherited copper disorder. The study has just begun; first results are expected in 2027.
-
Australian researchers give doctors a 'switch' to control immune cells as medicine
Researchers at WEHI in Melbourne have modified immune cells so that doctors can control them using two existing medications: one helps the cells grow, while the other inhibits them. The next step is a first study in humans.
-
Brain enzyme helps keep memory sharp in old age
Researchers in Boston have discovered an enzyme that the brain itself makes more of as we get older, and that seems to protect memory. In old mice, an extra dose kept memory in better shape. The next step is to find out whether this can also be done safely in humans.
