The Onpattro story and the clinical translation of nanomedicines containing nucleic acid-based drugs von 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.

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Advanced siRNA Designs Further Improve In Vivo Performance of GalNAc-siRNA Conjugates von Foster, Donald J., Brown, Christopher R., Shaikh, Sarfraz, Trapp, Casey, Schlegel, Mark K., Qian, Kun, Sehgal, Alfica, Rajeev, Kallanthottathil G., Jadhav, Vasant, Manoharan, Muthiah, Kuchimanchi, Satya, Maier, Martin A., Milstein, Stuart

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Selection of GalNAc-conjugated siRNAs with limited off-target-driven rat hepatotoxicity von Janas, Maja M., Schlegel, Mark K., Harbison, Carole E., Yilmaz, Vedat O., Jiang, Yongfeng, Parmar, Rubina, Zlatev, Ivan, Castoreno, Adam, Xu, Huilei, Shulga-Morskaya, Svetlana, Rajeev, Kallanthottathil G., Manoharan, Muthiah, Keirstead, Natalie D., Maier, Martin A., Jadhav, Vasant

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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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Maximizing the Potency of siRNA Lipid Nanoparticles for Hepatic Gene Silencing In Vivo von Jayaraman, Muthusamy, Ansell, Steven M., Mui, Barbara L., Tam, Ying K., Chen, Jianxin, Du, Xinyao, Butler, David, Eltepu, Laxman, Matsuda, Shigeo, Narayanannair, Jayaprakash K., Rajeev, Kallanthottathil G., Hafez, Ismail M., Akinc, Akin, Maier, Martin A., Tracy, Mark A., Cullis, Pieter R., Madden, Thomas D., Manoharan, Muthiah, Hope, Michael J.

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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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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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Rational design of cationic lipids for siRNA delivery von Semple, Sean C, Akinc, Akin, Chen, Jianxin, Sandhu, Ammen P, Mui, Barbara L, Cho, Connie K, Sah, Dinah W Y, Stebbing, Derrick, Crosley, Erin J, Yaworski, Ed, Hafez, Ismail M, Dorkin, J Robert, Qin, June, Lam, Kieu, Rajeev, Kallanthottathil G, Wong, Kim F, Jeffs, Lloyd B, Nechev, Lubomir, Eisenhardt, Merete L, Jayaraman, Muthusamy, Kazem, Mikameh, Maier, Martin A, Srinivasulu, Masuna, Weinstein, Michael J, Chen, Qingmin, Alvarez, Rene, Barros, Scott A, De, Soma, Klimuk, Sandra K, Borland, Todd, Kosovrasti, Verbena, Cantley, William L, Tam, Ying K, Manoharan, Muthiah, Ciufolini, Marco A, Tracy, Mark A, de Fougerolles, Antonin, MacLachlan, Ian, Cullis, Pieter R, Madden, Thomas D, Hope, Michael J

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Overcoming GNA/RNA base-pairing limitations using isonucleotides improves the pharmacodynamic activity of ESC+ GalNAc-siRNAs von Schlegel, Mark K, Matsuda, Shigeo, Brown, Christopher R, Harp, Joel M, Barry, Joseph D, Berman, Daniel, Castoreno, Adam, Schofield, Sally, Szeto, John, Manoharan, Muthiah, Charissé, Klaus, Egli, Martin, Maier, Martin A

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Small circular interfering RNAs (sciRNAs) as a potent therapeutic platform for gene-silencing von 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

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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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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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5′-(E)-Vinylphosphonate: A Stable Phosphate Mimic Can Improve the RNAi Activity of siRNA-GalNAc Conjugates von Parmar, Rubina, Willoughby, Jennifer L. S., Liu, Jingxuan, Foster, Donald J., Brigham, Benjamin, Theile, Christopher S., Charisse, Klaus, Akinc, Akin, Guidry, Erin, Pei, Yi, Strapps, Walter, Cancilla, Mark, Stanton, Matthew G., Rajeev, Kallanthottathil G., Sepp-Lorenzino, Laura, Manoharan, Muthiah, Meyers, Rachel, Maier, Martin A., Jadhav, Vasant

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Facile Synthesis, Geometry, and 2′-Substituent-Dependent in Vivo Activity of 5′‑(E)- and 5′‑(Z)‑Vinylphosphonate-Modified siRNA Conjugates von Parmar, Rubina Giare, Brown, Christopher R, Matsuda, Shigeo, Willoughby, Jennifer L. S, Theile, Christopher S, Charissé, Klaus, Foster, Donald J, Zlatev, Ivan, Jadhav, Vasant, Maier, Martin A, Egli, Martin, Manoharan, Muthiah, Rajeev, Kallanthottathil G

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Influence of Polyethylene Glycol Lipid Desorption Rates on Pharmacokinetics and Pharmacodynamics of siRNA Lipid Nanoparticles von Mui, Barbara L, Tam, Ying K, Jayaraman, Muthusamy, Ansell, Steven M, Du, Xinyao, Tam, Yuen Yi C, Lin, Paulo JC, Chen, Sam, Narayanannair, Jayaprakash K, Rajeev, Kallanthottathil G, Manoharan, Muthiah, Akinc, Akin, Maier, Martin A, Cullis, Pieter, Madden, Thomas D, Hope, Michael J

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Structural basis for the synergy of 4′- and 2′-modifications on siRNA nuclease resistance, thermal stability and RNAi activity von Harp, Joel M, Guenther, Dale C, Bisbe, Anna, Perkins, Lydia, Matsuda, Shigeo, Bommineni, Gopal R, Zlatev, Ivan, Foster, Donald J, Taneja, Nate, Charisse, Klaus, Maier, Martin A, Rajeev, Kallanthottathil G, Manoharan, Muthiah, Egli, Martin

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RNAi-mediated rheostat for dynamic control of AAV-delivered transgenes von 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

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RNA interference in the era of nucleic acid therapeutics von Jadhav, Vasant, Vaishnaw, Akshay, Fitzgerald, Kevin, Maier, Martin A.

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Structural Basis of Duplex Thermodynamic Stability and Enhanced Nuclease Resistance of 5′‑C‑Methyl Pyrimidine-Modified Oligonucleotides von Kel′in, Alexander V, Zlatev, Ivan, Harp, Joel, Jayaraman, Muthusamy, Bisbe, Anna, O′Shea, Jonathan, Taneja, Nate, Manoharan, Rajar M, Khan, Saeed, Charisse, Klaus, Maier, Martin A, Egli, Martin, Rajeev, Kallanthottathil G, Manoharan, Muthiah

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