Control of phosphorothioate stereochemistry substantially increases the efficacy of antisense oligonucleotides von Iwamoto, Naoki, Butler, David C D, Svrzikapa, Nenad, Mohapatra, Susovan, Zlatev, Ivan, Sah, Dinah W Y, Meena, Standley, Stephany M, Lu, Genliang, Apponi, Luciano H, Frank-Kamenetsky, Maria, Zhang, Jason Jingxin, Vargeese, Chandra, Verdine, Gregory L

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Effect of an RNA interference drug on the synthesis of proprotein convertase subtilisin/kexin type 9 (PCSK9) and the concentration of serum LDL cholesterol in healthy volunteers: a... von Fitzgerald, Kevin, Dr, Frank-Kamenetsky, Maria, PhD, Shulga-Morskaya, Svetlana, MSc, Liebow, Abigail, BS, Bettencourt, Brian R, PhD, Sutherland, Jessica E, PhD, Hutabarat, Renta M, PhD, Clausen, Valerie A, PhD, Karsten, Verena, PhD, Cehelsky, Jeffrey, MBA, Nochur, Saraswathy V, PhD, Kotelianski, Victor, MD, Horton, Jay, MD, Mant, Timothy, MD, Chiesa, Joseph, MD, Ritter, James, MD, Munisamy, Malathy, MD, Vaishnaw, Akshay K, MD, Gollob, Jared A, MD, Simon, Amy, MD

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The Scap/SREBP Pathway Is Essential for Developing Diabetic Fatty Liver and Carbohydrate-Induced Hypertriglyceridemia in Animals von Moon, Young-Ah, Liang, Guosheng, Xie, Xuefen, Frank-Kamenetsky, Maria, Fitzgerald, Kevin, Koteliansky, Victor, Brown, Michael S., Goldstein, Joseph L., Horton, Jay D.

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From noncoding variant to phenotype via SORT1 at the 1p13 cholesterol locus von Musunuru, Kiran, Strong, Alanna, Frank-Kamenetsky, Maria, Lee, Noemi E., Ahfeldt, Tim, Sachs, Katherine V., Li, Xiaoyu, Li, Hui, Kuperwasser, Nicolas, Ruda, Vera M., Pirruccello, James P., Muchmore, Brian, Prokunina-Olsson, Ludmila, Hall, Jennifer L., Schadt, Eric E., Morales, Carlos R., Lund-Katz, Sissel, Phillips, Michael C., Wong, Jamie, Cantley, William, Racie, Timothy, Ejebe, Kenechi G., Orho-Melander, Marju, Melander, Olle, Koteliansky, Victor, Fitzgerald, Kevin, Krauss, Ronald M., Cowan, Chad A., Kathiresan, Sekar, Rader, Daniel J.

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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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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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Small-molecule modulators of Hedgehog signaling: identification and characterization of Smoothened agonists and antagonists von Frank-Kamenetsky, Maria, Zhang, Xiaoyan M, Bottega, Steve, Guicherit, Oivin, Wichterle, Hynek, Dudek, Henryk, Bumcrot, David, Wang, Frank Y, Jones, Simon, Shulok, Janine, Rubin, Lee L, Porter, Jeffery A

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Activation of ER stress and mTORC1 suppresses hepatic sortilin-1 levels in obese mice von Ai, Ding, Baez, Juan M, Jiang, Hongfeng, Conlon, Donna M, Hernandez-Ono, Antonio, Frank-Kamenetsky, Maria, Milstein, Stuart, Fitzgerald, Kevin, Murphy, Andrew J, Woo, Connie W, Strong, Alanna, Ginsberg, Henry N, Tabas, Ira, Rader, Daniel J, Tall, Alan R

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CT visualization of blood pool in rats by using long-circulating, iodine-containing micelles von Torchilin, Vladimir P., Frank-Kamenetsky, Maria D., Wolf, Gerald L.

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Stable polymeric micelles: Lymphangiographic contrast media for gamma scintigraphy and magnetic resonance imaging von Trubetskoy, Vladimir S., Frank-Kamenetsky, Maria D., Whiteman, Kathleen R., Wolf, Gerald L., Torchilin, Vladimir P.

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Silencing of Lipid Metabolism Genes through IRE1α-Mediated mRNA Decay Lowers Plasma Lipids in Mice von So, Jae-Seon, Hur, Kyu Yeon, Tarrio, Margarite, Ruda, Vera, Frank-Kamenetsky, Maria, Fitzgerald, Kevin, Koteliansky, Victor, Lichtman, Andrew H., Iwawaki, Takao, Glimcher, Laurie H., Lee, Ann-Hwee

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Targeted Delivery of RNAi Therapeutics With Endogenous and Exogenous Ligand-Based Mechanisms von Akinc, Akin, Querbes, William, De, Soma, Qin, June, Frank-Kamenetsky, Maria, Jayaprakash, K. Narayanannair, Jayaraman, Muthusamy, Rajeev, Kallanthottathil G, Cantley, William L, Dorkin, J Robert, Butler, James S, Qin, LiuLiang, Racie, Timothy, Sprague, Andrew, Fava, Eugenio, Zeigerer, Anja, Hope, Michael J, Zerial, Marino, Sah, Dinah WY, Fitzgerald, Kevin, Tracy, Mark A, Manoharan, Muthiah, Koteliansky, Victor, Fougerolles, Antonin de, Maier, Martin A

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IRE1α activation protects mice against acetaminophen-induced hepatotoxicity von Hur, Kyu Yeon, So, Jae-Seon, Ruda, Vera, Frank-Kamenetsky, Maria, Fitzgerald, Kevin, Koteliansky, Victor, Iwawaki, Takao, Glimcher, Laurie H, Lee, Ann-Hwee

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Allele Selective Antisense Oligonucleotides for Reducing Mutant Huntingtin (P1.8-051) von Zhong, Zhong, Brown, Jeffrey M., Frank-Kamenetsky, Maria, Aklilu, Kidist, Dodart, Jean-Cosme, Berkovitch, Shaunna, Taborn, Kristin, Byrne, Michael, Iwamoto, Naoki, Vargeese, Chandra

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Stereopure Oligonucleotides for the Selective Silencing of Mutant Huntingtin (4703) von Dale, Elena, Frank-Kamenetsky, Maria, Taborn, Kristin, Aklilu, Kidist, Liu, Yuanjing, Berkovitch, Shaunna, Brown, Jeffrey, Kandasamy, Pachamuthu, Kumarasamy, Jayakanthan, Mathieu, Steven, Iwamoto, Naoki, Yin, Yuan, Yang, Hailin, Standley, Stephany, Favaloro, Frank, Longo, Kenneth, Byrne, Michael, Vargeese, Chandra

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Preclinical evaluation of stereopure antisense oligonucleotides for allele-selective lowering of mutant HTT von Iwamoto, Naoki, Liu, Yuanjing, Frank-Kamenetsky, Maria, Maguire, Abbie, Tseng, Wei Chou, Taborn, Kristin, Kothari, Nayantara, Akhtar, Ali, Bowman, Keith, Shelke, Juili Dilip, Lamattina, Anthony, Hu, Xiao Shelley, Jang, Hyun Gyung, Kandasamy, Pachamuthu, Liu, Fangjun, Longo, Ken, Looby, Richard, Meena, Metterville, Jake, Pan, Qianli, Purcell-Estabrook, Erin, Shimizu, Mamoru, Prakasha, Priyanka Shiva, Standley, Stephany, Upadhyay, Hansini, Yang, Hailin, Yin, Yuan, Zhao, Anderson, Francis, Christopher, Byrne, Mike, Dale, Elena, Verdine, Gregory L., Vargeese, Chandra

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Sa1465 - The Pharmacological Properties of APOC3 Antisense Oligonucleotides are Enhanced by Control of Stereochemistry von Apponi, Luciano, Iwamoto, Naoki, Frank-Kamenetsky, Maria, Yang, Hailin, Braun, Maurine, Shah, Anee, Yin, Yuan, Butler, David, Lu, Genliang, Menon, Sethu, Standley, Stephany, Aduda, Vincent, Bowman, Keith, Longo, Kenneth, Pittet, Lynnelle, Zhang, Jason, Zhong, Zhong, Vargeese, Chandra

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Selectivity and Biodistribution of WVE-120101, a Potential Antisense Oligonucleotide Therapy for the Treatment of Huntington’s Disease (S56.001) von Meena, M, Zboray, Lori, Svrzikapa, Nenad, Monteys, Alex Mas, Mohapatra, Susovan, Lonkar, Pallavi, Sokolovsk, Anna, Yang, Hailin, Iwamoto, Naoki, Butler, David, Bowman, Keith, Standley, Stephany, Shimizu, Mamoru, Frank-Kamenetsky, Maria, Francis, Chris, Davidson, Beverly, Vargeese, Chandra

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Gene Silencing Activity of siRNAs with a Ribo-difluorotoluyl Nucleotide von Xia, Jie, Noronha, Anne, Toudjarska, Ivanka, Li, Feng, Akinc, Akin, Braich, Ravi, Frank-Kamenetsky, Maria, Rajeev, Kallanthottathil G, Egli, Martin, Manoharan, Muthiah

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Abstract 165: RNAi Therapeutics Targeting PCSK9 and ANGPTL3 for Mixed Hyperlipidemia von Frank-Kamenetsky, Maria, Shulga-Morskaya, Svetlana, Liebow, Abigail, Racie, Tim, Milstein, Stuart, Kuchimanchi, Satya, Charisse, Klaus, Epstein-Barash, Hila, Fitzgerald, Kevin

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