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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Targeting of Antithrombin in Hemophilia A or B with RNAi Therapy von Pasi, K. John, Rangarajan, Savita, Georgiev, Pencho, Mant, Tim, Creagh, Michael D, Lissitchkov, Toshko, Bevan, David, Austin, Steve, Hay, Charles R, Hegemann, Inga, Kazmi, Rashid, Chowdary, Pratima, Gercheva-Kyuchukova, Liana, Mamonov, Vasily, Timofeeva, Margarita, Soh, Chang-Heok, Garg, Pushkal, Vaishnaw, Akshay, Akinc, Akin, Sørensen, Benny, Ragni, Margaret V

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MicroRNAs 103 and 107 regulate insulin sensitivity von Trajkovski, Mirko, Hausser, Jean, Soutschek, Jürgen, Bhat, Bal, Akin, Akinc, Zavolan, Mihaela, Heim, Markus H., Stoffel, Markus

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Image-based analysis of lipid nanoparticle–mediated siRNA delivery, intracellular trafficking and endosomal escape von Gilleron, Jerome, Querbes, William, Zeigerer, Anja, Borodovsky, Anna, Marsico, Giovanni, Schubert, Undine, Manygoats, Kevin, Seifert, Sarah, Andree, Cordula, Stöter, Martin, Epstein-Barash, Hila, Zhang, Ligang, Koteliansky, Victor, Fitzgerald, Kevin, Fava, Eugenio, Bickle, Marc, Kalaidzidis, Yannis, Akinc, Akin, Maier, Martin, Zerial, Marino

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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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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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Lipopeptide nanoparticles for potent and selective siRNA delivery in rodents and nonhuman primates von Dong, Yizhou, Love, Kevin T., Dorkin, J. Robert, Sirirungruang, Sasilada, Zhang, Yunlong, Chen, Delai, Bogorad, Roman L., Yin, Hao, Chen, Yi, Vegas, Arturo J., Alabi, Christopher A., Sahay, Gaurav, Olejnik, Karsten T., Wang, Weiheng, Schroeder, Avi, Lytton-Jean, Abigail K. R., Siegwart, Daniel J., Akinc, Akin, Barnes, Carmen, Barros, Scott A., Carioto, Mary, Fitzgerald, Kevin, Hettinger, Julia, Kumar, Varun, Novobrantseva, Tatiana I., Qin, June, Querbes, William, Koteliansky, Victor, Langer, Robert, Anderson, Daniel G.

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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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Exploring polyethylenimine-mediated DNA transfection and the proton sponge hypothesis von Akinc, Akin, Thomas, Mini, Klibanov, Alexander M., Langer, Robert

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Lipid-like materials for low-dose, in vivo gene silencing von Love, Kevin T, Mahon, Kerry P, Levins, Christopher G, Whitehead, Kathryn A, Querbes, William, Dorkin, J. Robert, Qin, June, Cantley, William, Qin, Liu Liang, Racie, Timothy, Frank-Kamenetsky, Maria, Yip, Ka Ning, Alvarez, Rene, Sah, Dinah W.Y, de Fougerolles, Antonin, Fitzgerald, Kevin, Koteliansky, Victor, Akinc, Akin, Langer, Robert, Anderson, Daniel G

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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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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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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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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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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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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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Shielding of Lipid Nanoparticles for siRNA Delivery: Impact on Physicochemical Properties, Cytokine Induction, and Efficacy von Kumar, Varun, Qin, June, Jiang, Yongfeng, Duncan, Richard G, Brigham, Benjamin, Fishman, Shannon, Nair, Jayaprakash K, Akinc, Akin, Barros, Scott A, Kasperkovitz, Pia V

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Influence of Cationic Lipid Composition on Gene Silencing Properties of Lipid Nanoparticle Formulations of siRNA in Antigen-Presenting Cells von Basha, Genc, Novobrantseva, Tatiana I, Rosin, Nicole, Tam, Yuen Yi C, Hafez, Ismail M, Wong, Matthew K, Sugo, Tsukasa, Ruda, Vera M, Qin, June, Klebanov, Boris, Ciufolini, Marco, Akinc, Akin, Tam, Ying K, Hope, Michael J, Cullis, Pieter R

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A Polymer Library Approach to Suicide Gene Therapy for Cancer von Anderson, Daniel G., Peng, Weidan, Akinc, Akin, Hossain, Naushad, Kohn, Anat, Padera, Robert, Langer, Robert, Sawicki, Janet A.

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