Antisense oligonucleotide treatment in a preterm infant with early-onset SCN2A developmental and epileptic encephalopathy
反义寡核苷酸治疗早产儿早期发作的SCN2A发育和癫痫性脑病
期刊:Nature Medicine
影响因子:50
doi:10.1038/s41591-025-03656-0
Wagner, Matias; Berecki, Géza; Fazeli, Walid; Nussbaum, Claudia; Flemmer, Andreas W; Frizzo, Silvana; Heer, Farina; Heinen, Florian; Horton, Robert; Jacotin, Henry; Motel, William; Spar, Brian; Klein, Christoph; Siegel, Corinna; Hübener, Christoph; Stöcklein, Sophia; Paolini, Marco; Staudt, Martin; Tacke, Moritz; Wolff, Markus; Petrou, Steven; Souza, Marcio; Borggraefe, Ingo