Angelman syndrome patient-derived neuron screen leads to clinical ASO rugonersen targeting UBE3A-ATS with long-lasting effect in monkeys.
安格曼综合征患者来源的神经元筛选产生了临床 ASO rugonersen，该药物靶向 UBE3A-ATS，在猴子身上具有持久疗效
期刊:Nucleic Acids Research
影响因子:13.1
doi:10.1093/nar/gkaf851
Jagasia Ravi, Bon Charlotte, Rasmussen Soren V, Badillo Solveig, Tehler Disa, Buchy Danièle, Berrera Marco, Prasad Megana, Chamberlain Stormy, Terrigno Marco, Pandya Nikhil J, Costa Veronica, Wang Congwei, Pedersen Lykke, Miller Meghan T, Erichsen Kamille Dumong, Joenson Lars, Patsch Christoph, Hipp Joerg F, Bonni Azad, Müller Lutz, Braendli-Baiocco Annamaria, Kremer Thomas, Koller Erich, Hoener Marius C
