COMPLEMENT COMPONENT C5 IRNA COMPOSITIONS AND METHODS OF USE THEREOF C5 IRNA von Martin MAIER M, Brian BETTENCOURT B, James BUTLER J, Kallanthottathil G. RAJEEV KG, Kevin FITZGERALD K, Satyanarayana KUCHIMANCHI S, Anna BORODOVSKY A, Donald FOSTER D, Klaus CHARISSE K, Muthiah MANOHARAN M

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Multivalent N‑Acetylgalactosamine-Conjugated siRNA Localizes in Hepatocytes and Elicits Robust RNAi-Mediated Gene Silencing von Nair, Jayaprakash K., Willoughby, Jennifer L. S., Chan, Amy, Charisse, Klaus, Alam, Md. Rowshon, Wang, Qianfan, Hoekstra, Menno, Kandasamy, Pachamuthu, Kel’in, Alexander V., Milstein, Stuart, Taneja, Nate, O’Shea, Jonathan, Shaikh, Sarfraz, Zhang, Ligang, van der Sluis, Ronald J., Jung, Michael E., Akinc, Akin, Hutabarat, Renta, Kuchimanchi, Satya, Fitzgerald, Kevin, Zimmermann, Tracy, van Berkel, Theo J. C., Maier, Martin A., Rajeev, Kallanthottathil G., Manoharan, Muthiah

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Impact of enhanced metabolic stability on pharmacokinetics and pharmacodynamics of GalNAc-siRNA conjugates von Nair, Jayaprakash K, Attarwala, Husain, Sehgal, Alfica, Wang, Qianfan, Aluri, Krishna, Zhang, Xuemei, Gao, Minggeng, Liu, Ju, Indrakanti, Ramesh, Schofield, Sally, Kretschmer, Philip, Brown, Christopher R, Gupta, Swati, Willoughby, Jennifer L S, Boshar, Julie A, Jadhav, Vasant, Charisse, Klaus, Zimmermann, Tracy, Fitzgerald, Kevin, Manoharan, Muthiah, Rajeev, Kallanthottathil G, Akinc, Akin, Hutabarat, Renta, Maier, Martin A

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Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs von Soutschek, Jürgen, Akinc, Akin, Bramlage, Birgit, Charisse, Klaus, Constien, Rainer, Donoghue, Mary, Elbashir, Sayda, Geick, Anke, Hadwiger, Philipp, Harborth, Jens, John, Matthias, Kesavan, Venkitasamy, Lavine, Gary, Pandey, Rajendra K, Racie, Timothy, Rajeev, Kallanthottathil G, Röhl, Ingo, Toudjarska, Ivanka, Wang, Gang, Wuschko, Silvio, Bumcrot, David, Koteliansky, Victor, Limmer, Stefan, Manoharan, Muthiah, Vornlocher, Hans-Peter

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An RNAi therapeutic targeting antithrombin to rebalance the coagulation system and promote hemostasis in hemophilia von Sehgal, Alfica, Barros, Scott, Ivanciu, Lacramioara, Cooley, Brian, Qin, June, Racie, Tim, Hettinger, Julia, Carioto, Mary, Jiang, Yongfeng, Brodsky, Josh, Prabhala, Harsha, Zhang, Xuemei, Attarwala, Husain, Hutabarat, Renta, Foster, Don, Milstein, Stuart, Charisse, Klaus, Kuchimanchi, Satya, Maier, Martin A, Nechev, Lubo, Kandasamy, Pachamuthu, Kel'in, Alexander V, Nair, Jayaprakash K, Rajeev, Kallanthottathil G, Manoharan, Muthiah, Meyers, Rachel, Sorensen, Benny, Simon, Amy R, Dargaud, Yesim, Negrier, Claude, Camire, Rodney M, Akinc, Akin

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Hepatocyte-Specific Delivery of siRNAs Conjugated to Novel Non-nucleosidic Trivalent N-Acetylgalactosamine Elicits Robust Gene Silencing in Vivo von Rajeev, Kallanthottathil G., Nair, Jayaprakash K., Jayaraman, Muthusamy, Charisse, Klaus, Taneja, Nate, O'Shea, Jonathan, Willoughby, Jennifer L. S., Yucius, Kristina, Nguyen, Tuyen, Shulga-Morskaya, Svetlana, Milstein, Stuart, Liebow, Abigail, Querbes, William, Borodovsky, Anna, Fitzgerald, Kevin, Maier, Martin A., Manoharan, Muthiah

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Evaluation of GalNAc-siRNA Conjugate Activity in Pre-clinical Animal Models with Reduced Asialoglycoprotein Receptor Expression von Willoughby, Jennifer L.S., Chan, Amy, Sehgal, Alfica, Butler, James S., Nair, Jayaprakash K., Racie, Tim, Shulga-Morskaya, Svetlana, Nguyen, Tuyen, Qian, Kun, Yucius, Kristina, Charisse, Klaus, van Berkel, Theo J.C., Manoharan, Muthiah, Rajeev, Kallanthottathil G., Maier, Martin A., Jadhav, Vasant, Zimmermann, Tracy S.

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siRNA Conjugates Carrying Sequentially Assembled Trivalent N-Acetylgalactosamine Linked Through Nucleosides Elicit Robust Gene Silencing In Vivo in Hepatocytes von Matsuda, Shigeo, Keiser, Kristofer, Nair, Jayaprakash K, Charisse, Klaus, Manoharan, Rajar M, Kretschmer, Philip, Peng, Chang G, V. Kel’in, Alexander, Kandasamy, Pachamuthu, Willoughby, Jennifer L.S, Liebow, Abigail, Querbes, William, Yucius, Kristina, Nguyen, Tuyen, Milstein, Stuart, Maier, Martin A, Rajeev, Kallanthottathil G, Manoharan, Muthiah

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Chirality Dependent Potency Enhancement and Structural Impact of Glycol Nucleic Acid Modification on siRNA von Schlegel, Mark K, Foster, Donald J, Kel’in, Alexander V, Zlatev, Ivan, Bisbe, Anna, Jayaraman, Muthusamy, Lackey, Jeremy G, Rajeev, Kallanthottathil G, Charissé, Klaus, Harp, Joel, Pallan, Pradeep S, Maier, Martin A, Egli, Martin, Manoharan, Muthiah

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Expanding RNAi therapeutics to extrahepatic tissues with lipophilic conjugates von 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

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Molecularly self-assembled nucleic acid nanoparticles for targeted in vivo siRNA delivery von Lee, Hyukjin, Lytton-Jean, Abigail K. R., Chen, Yi, Love, Kevin T., Park, Angela I., Karagiannis, Emmanouil D., Sehgal, Alfica, Querbes, William, Zurenko, Christopher S., Jayaraman, Muthusamy, Peng, Chang G., Charisse, Klaus, Borodovsky, Anna, Manoharan, Muthiah, Donahoe, Jessica S., Truelove, Jessica, Nahrendorf, Matthias, Langer, Robert, Anderson, Daniel G.

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Therapeutic RNAi targeting PCSK9 acutely lowers plasma cholesterol in rodents and LDL cholesterol in nonhuman primates von Frank-Kamenetsky, Maria, Grefhorst, Aldo, Anderson, Norma N, Racie, Timothy S, Bramlage, Birgit, Akinc, Akin, Butler, David, Charisse, Klaus, Dorkin, Robert, Fan, Yupeng, Gamba-Vitalo, Christina, Hadwiger, Philipp, Jayaraman, Muthusamy, John, Matthias, Jayaprakash, K. Narayanannair, Maier, Martin, Nechev, Lubomir, Rajeev, Kallanthottathil G, Read, Timothy, Röhl, Ingo, Soutschek, Jürgen, Tan, Pamela, Wong, Jamie, Wang, Gang, Zimmermann, Tracy, de Fougerolles, Antonin, Vornlocher, Hans-Peter, Langer, Robert, Anderson, Daniel G, Manoharan, Muthiah, Koteliansky, Victor, Horton, Jay D, Fitzgerald, Kevin

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5′-Morpholino modification of the sense strand of an siRNA makes it a more effective passenger von Kumar, Pawan, Parmar, Rubina G, Brown, Christopher R, Willoughby, Jennifer L. S, Foster, Donald J, Babu, I. Ramesh, Schofield, Sally, Jadhav, Vasant, Charisse, Klaus, Nair, Jayaprakash K, Rajeev, Kallanthottathil G, Maier, Martin A, Egli, Martin, Manoharan, Muthiah

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Biodegradable Lipids Enabling Rapidly Eliminated Lipid Nanoparticles for Systemic Delivery of RNAi Therapeutics von Maier, Martin A, Jayaraman, Muthusamy, Matsuda, Shigeo, Liu, Ju, Barros, Scott, Querbes, William, Tam, Ying K, Ansell, Steven M, Kumar, Varun, Qin, June, Zhang, Xuemei, Wang, Qianfan, Panesar, Sue, Hutabarat, Renta, Carioto, Mary, Hettinger, Julia, Kandasamy, Pachamuthu, Butler, David, Rajeev, Kallanthottathil G, Pang, Bo, Charisse, Klaus, Fitzgerald, Kevin, Mui, Barbara L, Du, Xinyao, Cullis, Pieter, Madden, Thomas D, Hope, Michael J, Manoharan, Muthiah, Akinc, Akin

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Versatile Site-Specific Conjugation of Small Molecules to siRNA Using Click Chemistry von Yamada, Takeshi, Peng, Chang Geng, Matsuda, Shigeo, Addepalli, Haripriya, Jayaprakash, K. Narayanannair, Alam, Md. Rowshon, Mills, Kathy, Maier, Martin A, Charisse, Klaus, Sekine, Mitsuo, Manoharan, Muthiah, Rajeev, Kallanthottathil G

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From bench to bedside: Improving the clinical safety of GalNAc–siRNA conjugates using seed-pairing destabilization von 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, Charissé, Klaus, Sepp-Lorenzino, Laura, Haslett, Patrick, Fitzgerald, Kevin, Jadhav, Vasant, Maier, Martin A

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Safety evaluation of 2′-deoxy-2′-fluoro nucleotides in GalNAc-siRNA conjugates von 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

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Molecularly self-assembled nucleic acid nanoparticles for targeted in vivo siRNA delivery von Lee, Hyukjin, Lytton-Jean, Abigail K. R., Chen, Yi, Love, Kevin T., Park, Angela I., Karagiannis, Emmanouil D., Sehgal, Alfica, Querbes, William, Zurenko, Christopher S., Jayaraman, Muthusamy, Peng, Chang G., Charisse, Klaus, Borodovsky, Anna, Manoharan, Muthiah, Donahoe, Jessica S., Truelove, Jessica, Nahrendorf, Matthias, Langer, Robert, Anderson, Daniel G.

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Sequence-Defined Oligomers from Hydroxyproline Building Blocks for Parallel Synthesis Applications von Kanasty, Rosemary L., Vegas, Arturo J., Ceo, Luke M., Maier, Martin, Charisse, Klaus, Nair, Jayaprakash K., Langer, Robert, Anderson, Daniel G.

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Modulation of thermal stability can enhance the potency of siRNA von Addepalli, Haripriya, Meena, Peng, Chang G, Wang, Gang, Fan, Yupeng, Charisse, Klaus, Jayaprakash, K Narayanannair, Rajeev, Kallanthottathil G, Pandey, Rajendra K, Lavine, Gary, Zhang, Ligang, Jahn-Hofmann, Kerstin, Hadwiger, Philipp, Manoharan, Muthiah, Maier, Martin A

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