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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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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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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A combinatorial library of lipid-like materials for delivery of RNAi therapeutics von Akinc, Akin, Zumbuehl, Andreas, Goldberg, Michael, Leshchiner, Elizaveta S, Busini, Valentina, Hossain, Naushad, Bacallado, Sergio A, Nguyen, David N, Fuller, Jason, Alvarez, Rene, Borodovsky, Anna, Borland, Todd, Constien, Rainer, de Fougerolles, Antonin, Dorkin, J Robert, Narayanannair Jayaprakash, K, Jayaraman, Muthusamy, John, Matthias, Koteliansky, Victor, Manoharan, Muthiah, Nechev, Lubomir, Qin, June, Racie, Timothy, Raitcheva, Denitza, Rajeev, Kallanthottathil G, Sah, Dinah W Y, Soutschek, Jürgen, Toudjarska, Ivanka, Vornlocher, Hans-Peter, Zimmermann, Tracy S, Langer, Robert, Anderson, Daniel G

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Mechanisms and optimization of in vivo delivery of lipophilic siRNAs von Wolfrum, Christian, Shi, Shuanping, Jayaprakash, K Narayanannair, Jayaraman, Muthusamy, Wang, Gang, Pandey, Rajendra K, Rajeev, Kallanthottathil G, Nakayama, Tomoko, Charrise, Klaus, Ndungo, Esther M, Zimmermann, Tracy, Koteliansky, Victor, Manoharan, Muthiah, Stoffel, Markus

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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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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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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 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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Investigating the pharmacodynamic durability of GalNAc–siRNA conjugates von 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

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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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Lipid Nanoparticles Improve Activity of Single-Stranded siRNA and Gapmer Antisense Oligonucleotides in Animals von Prakash, Thazha P, Lima, Walt F, Murray, Heather M, Elbashir, Sayda, Cantley, William, Foster, Don, Jayaraman, Muthusamy, Chappell, Alfred E, Manoharan, Muthiah, Swayze, Eric E, Crooke, Stanley T

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Systemic RNAi-mediated Gene Silencing in Nonhuman Primate and Rodent Myeloid Cells von Novobrantseva, Tatiana I, Borodovsky, Anna, Wong, Jamie, Klebanov, Boris, Zafari, Mohammad, Yucius, Kristina, Querbes, William, Ge, Pei, Ruda, Vera M, Milstein, Stuart, Speciner, Lauren, Duncan, Rick, Barros, Scott, Basha, Genc, Cullis, Pieter, Akinc, Akin, Donahoe, Jessica S, Narayanannair Jayaprakash, K, Jayaraman, Muthusamy, Bogorad, Roman L, Love, Kevin, Whitehead, Katie, Levins, Chris, Manoharan, Muthiah, Swirski, Filip K, Weissleder, Ralph, Langer, Robert, Anderson, Daniel G, de Fougerolles, Antonin, Nahrendorf, Matthias, Koteliansky, Victor

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Discovery of (+)-N-(3-Aminopropyl)-N-[1-(5-benzyl-3-methyl-4-oxo-[1,2]thiazolo[5,4-d]pyrimidin-6-yl)-2-methylpropyl]-4-methylbenzamide (AZD4877), a Kinesin Spindle Protein Inhibito... von Theoclitou, Maria-Elena, Aquila, Brian, Block, Michael H, Brassil, Patrick J, Castriotta, Lillian, Code, Erin, Collins, Michael P, Davies, Audrey M, Deegan, Tracy, Ezhuthachan, Jayachandran, Filla, Sandra, Freed, Ellen, Hu, Haiqing, Huszar, Dennis, Jayaraman, Muthusamy, Lawson, Deborah, Lewis, Paula M, Nadella, Murali V. P, Oza, Vibha, Padmanilayam, Maniyan, Pontz, Timothy, Ronco, Lucienne, Russell, Daniel, Whitston, David, Zheng, Xiaolan

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Cellular Topoisomerase I Inhibition and Antiproliferative Activity by MJ-III-65 (NSC 706744), an Indenoisoquinoline Topoisomerase I Poison von Antony, Smitha, Kohlhagen, Glenda, Agama, Keli, Jayaraman, Muthusamy, Cao, Shousong, Durrani, Farukh A., Rustum, Youcef M., Cushman, Mark, Pommier, Yves

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Synthesis of New Dihydroindeno[1,2-c]isoquinoline and Indenoisoquinolinium Chloride Topoisomerase I Inhibitors Having High in Vivo Anticancer Activity in the Hollow Fiber Animal Mo... von Jayaraman, Muthusamy, Fox, Brian M, Hollingshead, Melinda, Kohlhagen, Glenda, Pommier, Yves, Cushman, Mark

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Synthesis of Cytotoxic Indenoisoquinoline Topoisomerase I Poisons von Strumberg, Dirk, Pommier, Yves, Paull, Kenneth, Jayaraman, Muthusamy, Nagafuji, Pamela, Cushman, Mark

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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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The Nonclinical Disposition and Pharmacokinetic/Pharmacodynamic Properties of N -Acetylgalactosamine-Conjugated Small Interfering RNA Are Highly Predictable and Build Confidence in... von McDougall, Robin, Ramsden, Diane, Agarwal, Sagar, Agarwal, Saket, Aluri, Krishna, Arciprete, Michael, Brown, Christopher, Castellanos-Rizaldos, Elena, Charisse, Klaus, Chong, Saeho, Cichocki, Joseph, Fitzgerald, Kevin, Goel, Varun, Gu, Yongli, Guenther, Dale, Habtemariam, Bahru, Jadhav, Vasant, Janas, Maja, Jayaraman, Muthusamy, Kurz, Jeffrey, Li, Jing, Liu, Ju, Liu, Xiumin, Liou, Steven, Maclauchlin, Chris, Maier, Martin, Manoharan, Muthiah, Nair, Jayaprakash K, Robbie, Gabriel, Schmidt, Karyn, Smith, Peter, Theile, Christopher, Vaishnaw, Akshay, Waldron, Scott, Xu, Yuanxin, Zhang, Xuemei, Zlatev, Ivan, Wu, Jing-Tao

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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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