An orally available, brain penetrant, small molecule lowers huntingtin levels by enhancing pseudoexon inclusion
一种口服、可渗透大脑的小分子通过增强伪外显子的插入来降低亨廷顿蛋白的水平
期刊:Nature Communications
影响因子:14.7
doi:10.1038/s41467-022-28653-6
Caroline Gubser Keller #, Youngah Shin #, Alex Mas Monteys #, Nicole Renaud, Martin Beibel, Natalia Teider, Thomas Peters, Thomas Faller, Sophie St-Cyr, Judith Knehr, Guglielmo Roma, Alejandro Reyes, Marc Hild, Dmitriy Lukashev, Diethilde Theil, Natalie Dales, Jang-Ho Cha, Beth Borowsky, Ricardo Dol