NeurosciencebioRxivPreprint
Angelman syndrome patient neuron screen identifies a potent and selective clinical ASO targeting UBE3A-ATS with long lasting effect in cynomolgus monkey
Ravi Jagasia, Charlotte Bon, Soren V. Rasmussen…·Marius Christian Hoener
The paper identifies a potent ASO targeting UBE3A-ATS in Angelman syndrome patient neurons, showing long-lasting effects in cynomolgus monkeys.