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Improving the potency prediction for chemically modified siRNAs through insights from molecular modeling of individual sequence positions Kliuchnikov, Evgenii; Maksudov, Farkhad; Zuber, Jeffrey; Hyde, Sarah; Castoreno, Adam; Waldron, Scott; Schlegel, Mark K.; Marx, Kenneth A.; Maier, Martin A.; Barsegov, Valeri 分享 收藏
High resistance barrier and prophylactic protection in preclinical models of SARS-CoV-2 with two siRNA combination Anglero-Rodriguez, Yesseinia, I; Lempp, Florian A.; Subramanian, Megha; Mcininch, James; Schlegel, Mark K.; Bohan, Dana; Wong, Emily; Brown, Christopher R.; Foster, Donald J.; Castoreno, Adam B.; Nguyen, Tuyen; Cuffe, Dara; Montiel-Ruiz, Martin; Kaiser, Hannah; Sahakyan, Anna; Spreafico, Roberto; Morskaya, Svetlana Shulga; Barry, Joseph D.; Berman, Daniel; Zhang, Ligang; Lefebvre, Stephanie; Kasper, Anne; Racie, Timothy; Weddle, Diann; Mobley, Melissa; Wassarman, Kelly; Bisbe, Anna; Zlatev, Ivan; Rogers, Arlin; Nechev, Lubomir; Dybowski, Joseph; Chong, Saeho; Nair, Jayaprakash; Simon, Amy; Sloan, Kevin; Hwang, Seungmin; Virgin, Herbert W.; Fitzgerald, Kevin; Maier, Martin A.; Hinkle, Gregory; Hebner, Christy M.; Akinc, Akin; Jadhav, Vasant 分享 收藏
Evaluating the oral delivery of GalNAc-conjugated siRNAs in rodents and non-human primates Yu, Mikyung; Qin, June; Liu, Xiumin; Ramsden, Diane; Williams, Brian; Zlatev, Ivan; Guenther, Dale; Matsuda, Shigeo; Tymon, Roxanne; Darcy, Justin; Wong, Catrina; Tsung, Jamie; Zawaneh, Peter; Chong, Saeho; Theile, Christopher S.; Taneja, Nathan; Rogers, Arlin; Liu, Ju; Castellanos-Rizaldos, Elena; Bond, Sarah; So, Kawai; Denoncourt, Jason; Castoreno, Adam; Manoharan, Muthiah; Wu, Jing-Tao; Fitzgerald, Kevin; Maier, Martin A.; Jadhav, Vasant; Nair, Jayaprakash K. 分享 收藏
RNAi mediated inhibition of beta-catenin demonstrates anti-tumor efficacy and immune microenvironment modulation in preclinical hepatocellular carcinoma models Dadali, Tulin; Miller, Spencer; Saawant, Gaurav; Abbott, Stephen; Yu, Mikyung; Morskaya, Svetlana; Lehrich, Brandon; Monga, Satdarshan; Lau, Gloria; Maier, Martin; Broom, Wendy 分享 收藏
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RNAi-mediated rheostat for dynamic control of AAV-delivered transgenes Subramanian, Megha; McIninch, James; Zlatev, Ivan; Schlegel, Mark K.; Kaittanis, Charalambos; Nguyen, Tuyen; Agarwal, Saket; Racie, Timothy; Alvarado, Martha Arbaiza; Wassarman, Kelly; Collins, Thomas S.; Chickering, Tyler; Brown, Christopher R.; Schmidt, Karyn; Castoreno, Adam B.; Shulga-Morskaya, Svetlana; Stamenova, Elena; Buckowing, Kira; Berman, Daniel; Barry, Joseph D.; Bisbe, Anna; Maier, Martin A.; Fitzgerald, Kevin; Jadhav, Vasant 分享 收藏
Metabolically Stable Anomeric Linkages Containing GalNAc-siRNA Conjugates: An Interplay among ASGPR, Glycosidase, and RISC Pathways Kandasamy, Pachamuthu; Mori, Shohei; Matsuda, Shigeo; Erande, Namrata; Datta, Dhrubajyoti; Willoughby, Jennifer L. S.; Taneja, Nate; O'Shea, Jonathan; Bisbe, Anna; Manoharan, Rajar M.; Yucius, Kristina; Nguyen, Tuyen; Indrakanti, Ramesh; Gupta, Swati; Gilbert, Jason A.; Racie, Tim; Chan, Amy; Liu, Ju; Hutabarat, Renta; Nair, Jayaprakash K.; Charisse, Klaus; Maier, Martin A.; Rajeev, Kallanthottathil G.; Egli, Martin; Manoharan, Muthiah 分享 收藏
Role of a Magic Methyl: 2'-Deoxy-2'-a-F-2'-β-C-methyl Pyrimidine Nucleotides Modulate RNA Interference Activity through Synergy with 5'-Phosphate Mimics and Mitigation of Off-Target Effects Guenther, Dale C.; Mori, Shohei; Matsuda, Shigeo; Gilbert, Jason A.; Willoughby, Jennifer L. S.; Hyde, Sarah; Bisbe, Anna; Jiang, Yongfeng; Agarwal, Saket; Madaoui, Mimouna; Janas, Maja M.; Charisse, Klaus; Maier, Martin A.; Egli, Martin; Manoharan, Muthiah 分享 收藏
From bench to bedside: Improving the clinical safety of GalNAc-siRNA conjugates using seed-pairing destabilization Schlegel, Mark K.; Janas, Maja M.; Jiang, Yongfeng; Barry, Joseph D.; Davis, Wendell; Agarwal, Saket; Berman, Daniel; Brown, Christopher R.; Castoreno, Adam; LeBlanc, Sarah; Liebow, Abigail; Mayo, Tara; Milstein, Stuart; Nguyen, Tuyen; Shulga-Morskaya, Svetlana; Hyde, Sarah; Schofield, Sally; Szeto, John; Woods, Lauren Blair; Yilmaz, Vedat O.; Manoharan, Muthiah; Egli, Martin; Charisse, Klaus; Sepp-Lorenzino, Laura; Haslett, Patrick; Fitzgerald, Kevin; Jadhav, Vasant; Maier, Martin A. 分享 收藏
Expanding RNAi therapeutics to extrahepatic tissues with lipophilic conjugates 用亲脂结合物将RNAi疗法扩展到肝外组织 Brown, Kirk M.; Nair, Jayaprakash K.; Janas, Maja M.; Anglero-Rodriguez, Yesseinia I.; Dang, Lan T. H.; Peng, Haiyan; Theile, Christopher S.; Castellanos-Rizaldos, Elena; Brown, Christopher; Foster, Donald; Kurz, Jeffrey; Allen, Jeffrey; Maganti, Rajanikanth; Li, Jing; Matsuda, Shigeo; Stricos, Matthew; Chickering, Tyler; Jung, Michelle; Wassarman, Kelly; Rollins, Jeff; Woods, Lauren; Kelin, Alex; Guenther, Dale C.; Mobley, Melissa W.; Petrulis, John; McDougall, Robin; Racie, Timothy; Bombardier, Jessica; Cha, Diana; Agarwal, Saket; Johnson, Lei; Jiang, Yongfeng; Lentini, Scott; Gilbert, Jason; Nguyen, Tuyen; Chigas, Samantha; LeBlanc, Sarah; Poreci, Urjana; Kasper, Anne; Rogers, Arlin B.; Chong, Saeho; Davis, Wendell; Sutherland, Jessica E.; Castoreno, Adam; Milstein, Stuart; Schlegel, Mark K.; Zlatev, Ivan; Charisse, Klaus; Keating, Mark; Manoharan, Muthiah; Fitzgerald, Kevin; Wu, Jing-Tao; Maier, Martin A.; Jadhav, Vasant 分享 收藏
Overcoming GNA/RNA base-pairing limitations using isonucleotides improves the pharmacodynamic activity of ESC plus GalNAc-siRNAs Schlegel, Mark K.; Matsuda, Shigeo; Brown, Christopher R.; Harp, Joel M.; Barry, Joseph D.; Berman, Daniel; Castoreno, Adam; Schofield, Sally; Szeto, John; Manoharan, Muthiah; Charisse, Klaus; Egli, Martin; Maier, Martin A. 分享 收藏
Small circular interfering RNAs (sciRNAs) as a potent therapeutic platform for gene-silencing Jahns, Hartmut; Degaonkar, Rohan; Podbevsek, Peter; Gupta, Swati; Bisbe, Anna; Aluri, Krishna; Szeto, John; Kumar, Pawan; LeBlanc, Sarah; Racie, Tim; Brown, Christopher R.; Castoreno, Adam; Guenther, Dale C.; Jadhav, Vasant; Maier, Martin A.; Plavec, Janez; Egli, Martin; Manoharan, Muthiah; Zlatev, Ivan 分享 收藏
Chirality matters: stereo-defined phosphorothioate linkages at the termini of small interfering RNAs improve pharmacology in vivo Jahns, Hartmut; Taneja, Nate; Willoughby, Jennifer L. S.; Akabane-Nakata, Masaaki; Brown, Christopher R.; Nguyen, Tuyen; Bisbe, Anna; Matsuda, Shigeo; Hettinger, Matt; Manoharan, Rajar M.; Rajeev, Kallanthottathil G.; Maier, Martin A.; Zlatev, Ivan; Charisse, Klaus; Egli, Martin; Manoharan, Muthiah 分享 收藏
Centyrin ligands for extrahepatic delivery of siRNA Klein, Donna; Goldberg, Shalom; Theile, Christopher S.; Dambra, Richard; Haskell, Kathleen; Kuhar, Elise; Lin, Tricia; Parmar, Rubina; Manoharan, Muthiah; Richter, Mark; Wu, Meizhen; Zarazowski, Jeannine Mendrola; Jadhav, Vasant; Maier, Martin A.; Sepp-Lorenzino, Laura; O'Neil, Karyn; Dudkin, Vadim 分享 收藏
Investigating the pharmacodynamic durability o f GalNAc-siRNA conjugates Brown, Christopher R.; Gupta, Swati; Qin, June; Racie, Timothy; He, Guo; Lentini, Scott; Malone, Ryan; Yu, Mikyung; Matsuda, Shigeo; Shulga-Morskaya, Svetlana; Nair, Anil, V; Theile, Christopher S.; Schmidt, Karyn; Shahraz, Azar; Goel, Varun; Parmar, Rubina G.; Zlatev, Ivan; Schlegel, Mark K.; Nair, Jayaprakash K.; Jayaraman, Muthusamy; Manoharan, Muthiah; Brown, Dennis; Maier, Martin A.; Jadhav, Vasant 分享 收藏
Generation and validation of structurally defined antibody-siRNA conjugates Nanna, Alex R.; Kel'in, Alexander, V; Theile, Christopher; Pierson, Justin M.; Voo, Zhi Xiang; Garg, Ashish; Nair, Jayaprakash K.; Maier, Martin A.; Fitzgerald, Kevin; Rader, Christoph 分享 收藏
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The Onpattro story and the clinical translation of nanomedicines containing nucleic acid-based drugs 含核酸药物的纳米药物的Onpattro故事和临床翻译 Akinc, Akin; Maier, Martin A.; Manoharan, Muthiah; Fitzgerald, Kevin; Jayaraman, Muthusamy; Barros, Scott; Ansell, Steven; Du, Xinyao; Hope, Michael J.; Madden, Thomas D.; Mui, Barbara L.; Semple, Sean C.; Tam, Ying K.; Ciufolini, Marco; Witzigmann, Dominik; Kulkarni, Jayesh A.; van der Meel, Roy; Cullis, Pieter R. 分享 收藏
Safety evaluation of 2-deoxy-2-fluoro nucleotides in GalNAc-siRNA conjugates Janas, Maja M.; Zlatev, Ivan; Liu, Ju; Jiang, Yongfeng; Barros, Scott A.; Sutherland, Jessica E.; Davis, Wendell P.; Liu, Jingxuan; Brown, Christopher R.; Liu, Xiumin; Schlegel, Mark K.; Blair, Lauren; Zhang, Xuemei; Das, Biplab; Tran, Chris; Aluri, Krishna; Li, Jing; Agarwal, Saket; Indrakanti, Ramesh; Charisse, Klaus; Nair, Jayaprakash; Matsuda, Shigeo; Rajeev, Kallanthottathil G.; Zimmermann, Tracy; Sepp-Lorenzino, Laura; Xu, Yuanxin; Akinc, Akin; Fitzgerald, Kevin; Vaishnaw, Akshay K.; Smith, Peter F.; Manoharan, Muthiah; Jadhav, Vasant; Wu, Jing-Tao; Maier, Martin A. 分享 收藏
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