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, Solveig Badillo, Disa Tehler, Danièle Buchy, Marco Berrera, Megana K. Prasad, Marco Terrigno, Nikhil Janak Pandya, Veronica Costa, Congwei Wang, Lykke Pedersen, Meghan T. Miller, Kamille Dumong Erichsen, , Marius Christian Hoener
The paper identifies a potent ASO targeting UBE3A-ATS in Angelman syndrome patient neurons, showing long-lasting effects in cynomolgus monkeys.
bioRxiv · Jun 9, 2022 · PreprintNeuroscience