The nucleobase guanine at the 3'-terminus of oligonucleotide RGLS4326 drives off-target AMPAR inhibition and CNS toxicity
寡核苷酸RGLS4326 3'末端的核碱基鸟嘌呤导致脱靶AMPA受体抑制和中枢神经系统毒性
期刊:Nature Communications
影响因子:15.7
doi:10.1038/s41467-025-65799-5
Valencia, Tania; Yen, Laura Y; Berman, Cindy; Vincent, Thomas; Davis, Scott; Varrone, Francesca; Huang, Jianfeng; Mastroianni, Jessica; Carlson, Morgan; Owen, Tate; Kamel, Amin; Drygin, Denis; Kinberger, Garth A; Gangwar, Shanti Pal; Yelshanskaya, Maria V; Ridley, John; Kirby, Robert; Alvarez, Jesus; Lakhia, Ronak; Patel, Vishal; Sobolevsky, Alexander I; Lee, Edmund C
