未登录 分享 收藏
FORCE platform overcomes barriers of oligonucleotide delivery to muscle and corrects myotonic dystrophy features in preclinical models Weeden, Timothy; Picariello, Tyler; Quinn, Brendan; Spring, Sean; Shen, Pei-Yi; Qiu, Qifeng; Vieira, Benjamin F.; Schlaefke, Lydia; Russo, Ryan J.; Chang, Ya-An; Cui, Jin; Yao, Monica; Wen, Aiyun; Hsia, Nelson; Evron, Tama; Ovington, Katy; Tsai, Pei-Ni; Yoder, Nicholas; Lan, Bo; Venkatesan, Reshmii; Hall, John; Desjardins, Cody A.; Qatanani, Mo; Hilderbrand, Scott; Najim, John; Tang, Zhenzhi; Tanner, Matthew K.; Subramanian, Romesh; Thornton, Charles A.; Ibraghimov-Beskrovnaya, Oxana; Zanotti, Stefano 分享 收藏
Enhanced exon skipping and prolonged dystrophin restoration achieved by TfR1-targeted delivery of antisense oligonucleotide using FORCE conjugation in mdx mice Desjardins, Cody A.; Yao, Monica; Hall, John; O'Donnell, Emma; Venkatesan, Reshmii; Spring, Sean; Wen, Aiyun; Hsia, Nelson; Shen, Peiyi; Russo, Ryan; Lan, Bo; Picariello, Tyler; Tang, Kim; Weeden, Timothy; Zanotti, Stefano; Subramanian, Romesh; Ibraghimov-Beskrovnaya, Oxana 分享 收藏
Systemic Alanine Glyoxylate Aminotransferase mRNA Improves Glyoxylate Metabolism in a Mouse Model of Primary Hyperoxaluria Type 1 系统性丙氨酸乙醛酸氨基转移酶mRNA改善原发性高草酸尿症1型小鼠模型中的乙醛酸代谢 Kukreja, Anjli; Lasaro, Melissa; Cobaugh, Christian; Forbes, Chris; Tang, Jian-Ping; Gao, Xiang; Martin-Higueras, Cristina; Pey, Angel L.; Salido, Eduardo; Sobolov, Susan; Subramanian, Romesh R. 分享 收藏
分享 收藏
分享 收藏
分享 收藏
分享 收藏
分享 收藏
分享 收藏
Uptake, Efficacy, and Systemic Distribution of Naked, Inhaled Short Interfering RNA (siRNA) and Locked Nucleic Acid (LNA) Antisense Moschos, Sterghios A.; Frick, Manfred; Taylor, Bruce; Turnpenny, Paul; Graves, Helen; Spink, Karen G.; Brady, Kevin; Lamb, David; Collins, David; Rockel, Thomas D.; Weber, Markus; Lazari, Ovadia; Perez-Tosar, Luis; Fancy, Sally A.; Lapthorn, Chris; Green, Martin X.; Evans, Steve; Selby, Matthew; Jones, Gareth; Jones, Lyn; Kearney, Sarah; Mechiche, Houria; Gikunju, Diana; Subramanian, Romesh; Uhlmann, Eugen; Jurk, Marion; Vollmer, Joerg; Ciaramella, Giuseppe; Yeadon, Michael 分享 收藏
分享 收藏
分享 收藏
分享 收藏
分享 收藏
分享 收藏
分享 收藏
分享 收藏
分享 收藏
分享 收藏