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Modified unlocked nucleic acid (MUNA) mitigates off-target effects of small interfering RNAs 改性未锁定核酸（MUNA）可减轻小干扰RNA的脱靶效应 Mori, Shohei; Datta, Dhrubajyoti; Perkins, Lydia; Jung, Michelle; Woods, Lauren Blair; Eaton, Alex; Qin, June; Racie, Tim; Kim, MaryBeth; Guenther, Dale C.; Castoreno, Adam; Schlegel, Mark K.; Charisse, Klaus; Egli, Martin; Matsuda, Shigeo; Manoharan, Muthiah 分享 收藏
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
In vivo expansion of gene-targeted hepatocytes through transient inhibition of an essential gene 通过抑制一个必需基因实现体内基因靶向肝细胞的扩增 De Giorgi, Marco; Park, So Hyun; Castoreno, Adam; Cao, Mingming; Hurley, Ayrea; Saxena, Lavanya; Chuecos, Marcel A.; Walkey, Christopher J.; Doerfler, Alexandria M.; Furgurson, Mia N.; Ljungberg, M. Cecilia; Patel, Kalyani R.; Hyde, Sarah; Chickering, Tyler; Lefebvre, Stephanie; Wassarman, Kelly; Miller, Patrick; Qin, June; Schlegel, Mark K.; Zlatev, Ivan; Han, Jun; Beeton, Christine; Li, Rich Gang; Kim, Jong; Martin, James F.; Bissig, Karl-Dimiter; Jadhav, Vasant; Bao, Gang 分享 收藏
Expanding Conjugate Space of RNAi Therapeutics: Ligand at the 3′ End of the Antisense Strand Achieves Uncompromised In Vivo Potency and Efficacy and Reveals Interactions with the Argonaute-2 PAZ Domain 扩展RNAi疗法共轭空间：反义链3′端的配体实现了无妥协的在体效价和疗效，并揭示了与Argonaute-2 PAZ结构域的相互作用 Das, Rajat S.; Datta, Dhrubajyoti; Brown, Christopher R.; Gilbert, Jason A.; Chan, Amy; Willoughby, Jennifer; Gupta, Swati; Kim, Marybeth; Degaonkar, Rohan; Racie, Tim; Lei, Li; Schlegel, Mark K.; Castoreno, Adam; Charisse, Klaus; Rajeev, Kallanthottathil G.; Egli, Martin; Manoharan, Muthiah 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
Shorter Is Better: The a-(l)-Threofuranosyl Nucleic Acid Modification Improves Stability, Potency, Safety, and Ago2 Binding and Mitigates Off-Target Effects of Small Interfering RNAs Matsuda, Shigeo; Bala, Saikat; Liao, Jen-Yu; Datta, Dhrubajyoti; Mikami, Atsushi; Woods, Lauren; Harp, Joel M.; Gilbert, Jason A.; Bisbe, Anna; Manoharan, Rajar M.; Kim, MaryBeth; Theile, Christopher S.; Guenther, Dale C.; Jiang, Yongfeng; Agarwal, Saket; Maganti, Rajanikanth; Schlegel, Mark K.; Zlatev, Ivan; Charisse, Klaus; Rajeev, Kallanthottathil G.; Castoreno, Adam; Egli, Martin; Janas, Maja M.; Egli, Martin; Chaput, John C.; Manoharan, Muthiah 分享 收藏
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
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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
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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
siRNAs containing 2′-fluorinated Northern-methanocarbacyclic (2′-F-NMC) nucleotides: in vitro and in vivo RNAi activity and inability of mitochondrial polymerases to incorporate 2′-F-NMC NTPs Akabane-Nakata, Masaaki; Erande, Namrata D.; Kumar, Pawan; Degaonkar, Rohan; Gilbert, Jason A.; Qin, June; Mendez, Martha; Woods, Lauren Blair; Jiang, Yongfeng; Janas, Maja M.; O'Flaherty, Derek K.; Zlatev, Ivan; Schlegel, Mark K.; Matsuda, Shigeo; Egli, Martin; Manoharan, Muthiah 分享 收藏
Synthesis, chirality-dependent conformational and biological properties of siRNAs containing 5′-(R)- and 5′-(S)-C-methyl-guanosine Mikami, Atsushi; Erande, Namrata; Matsuda, Shigeo; Kel'in, Alexander; Woods, Lauren Blair; Chickering, Tyler; Pallan, Pradeep S.; Schlegel, Mark K.; Zlatev, Ivan; Egli, Martin; Manoharan, Muthiah 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
Chimeric siRNAs with chemically modified pentofuranose and hexopyranose nucleotides: altritol-nucleotide (ANA) containing GalNAc-siRNA conjugates: in vitro and in vivo RNAi activity and resistance to 5′-exonuclease Kumar, Pawan; Degaonkar, Rohan; Guenther, Dale C.; Abramov, Mikhail; Schepers, Guy; Capobianco, Marie; Jiang, Yongfeng; Harp, Joel; Kaittanis, Charalambos; Janas, Maja M.; Castoreno, Adam; Zlatev, Ivan; Schlegel, Mark K.; Herdewijn, Piet; Egli, Martin; Manoharan, Muthiah 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
Microbe-focused glycan array screening platform Geissner, Andreas; Reinhardt, Anika; Rademacher, Christoph; Johannssen, Timo; Monteiro, Joao; Lepenies, Bernd; Thepaut, Michel; Fieschi, Franck; Mrazkova, Jana; Wimmerova, Michaela; Schuhmacher, Frank; Goetze, Sebastian; Gruenstein, Dan; Guo, Xiaoqiang; Hahm, Heung Sik; Kandasamy, Jeyakumar; Leonori, Daniele; Martin, Christopher E.; Parameswarappa, Sharavathi G.; Pasari, Sandip; Schlegel, Mark K.; Tanaka, Hidenori; Xiao, Guozhi; Yang, You; Pereira, Claney L.; Anish, Chakkumkal; Seeberger, Peter H. 分享 收藏
An investigational RNAi therapeutic targeting Factor XII (ALN-F12) for the treatment of hereditary angioedema Liu, Jingxuan; Qin, June; Borodovsky, Anna; Racie, Timothy; Castoreno, Adam; Schlegel, Mark; Maier, Martin A.; Zimmerman, Tracy; Fitzgerald, Kevin; Butler, James; Akinc, Akin 分享 收藏
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