Variant-selective stereopure oligonucleotides protect against pathologies associated with C9orf72-repeat expansion in preclinical models von Liu, Yuanjing, Dodart, Jean-Cosme, Tran, Helene, Berkovitch, Shaunna, Braun, Maurine, Byrne, Michael, Durbin, Ann F., Hu, Xiao Shelley, Iwamoto, Naoki, Jang, Hyun Gyung, Kandasamy, Pachamuthu, Liu, Fangjun, Longo, Kenneth, Ruschel, Jörg, Shelke, Juili, Yang, Hailin, Yin, Yuan, Donner, Amy, Zhong, Zhong, Vargeese, Chandra, Brown, Robert H.

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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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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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Synthetic Routes to Thiooligosaccharides and Thioglycopeptides von Pachamuthu, Kandasamy, Schmidt, Richard R

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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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Endogenous ADAR-mediated RNA editing in non-human primates using stereopure chemically modified oligonucleotides von Monian, Prashant, Shivalila, Chikdu, Lu, Genliang, Shimizu, Mamoru, Boulay, David, Bussow, Karley, Byrne, Michael, Bezigian, Adam, Chatterjee, Arindom, Chew, David, Desai, Jigar, Favaloro, Frank, Godfrey, Jack, Hoss, Andrew, Iwamoto, Naoki, Kawamoto, Tomomi, Kumarasamy, Jayakanthan, Lamattina, Anthony, Lindsey, Amber, Liu, Fangjun, Looby, Richard, Marappan, Subramanian, Metterville, Jake, Murphy, Ronelle, Rossi, Jeff, Pu, Tom, Bhattarai, Bijay, Standley, Stephany, Tripathi, Snehlata, Yang, Hailin, Yin, Yuan, Yu, Hui, Zhou, Cong, Apponi, Luciano H., Kandasamy, Pachamuthu, Vargeese, Chandra

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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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Impact of guanidine-containing backbone linkages on stereopure antisense oligonucleotides in the CNS von Kandasamy, Pachamuthu, Liu, Yuanjing, Aduda, Vincent, Akare, Sandheep, Alam, Rowshon, Andreucci, Amy, Boulay, David, Bowman, Keith, Byrne, Michael, Cannon, Megan, Chivatakarn, Onanong, Shelke, Juili Dilip, Iwamoto, Naoki, Kawamoto, Tomomi, Kumarasamy, Jayakanthan, Lamore, Sarah, Lemaitre, Muriel, Lin, Xuena, Longo, Kenneth, Looby, Richard, Marappan, Subramanian, Metterville, Jake, Mohapatra, Susovan, Newman, Bridget, Paik, Ik-Hyeon, Patil, Saurabh, Purcell-Estabrook, Erin, Shimizu, Mamoru, Shum, Pochi, Standley, Stephany, Taborn, Kris, Tripathi, Snehlata, Yang, Hailin, Yin, Yuan, Zhao, Xiansi, Dale, Elena, Vargeese, Chandra

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Impact of stereopure chimeric backbone chemistries on the potency and durability of gene silencing by RNA interference von Liu, Wei, Iwamoto, Naoki, Marappan, Subramanian, Luu, Khoa, Tripathi, Snehlata, Purcell-Estabrook, Erin, Shelke, Juili Dilip, Shah, Himali, Lamattina, Anthony, Pan, Qianli, Schrand, Brett, Favaloro, Frank, Bedekar, Mugdha, Chatterjee, Arindom, Desai, Jigar, Kawamoto, Tomomi, Lu, Genliang, Metterville, Jake, Samaraweera, Milinda, Prakasha, Priyanka Shiva, Yang, Hailin, Yin, Yuan, Yu, Hui, Giangrande, Paloma H, Byrne, Michael, Kandasamy, Pachamuthu, Vargeese, Chandra

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Rational design of base, sugar and backbone modifications improves ADAR-mediated RNA editing von Lu, Genliang, Shivalila, Chikdu, Monian, Prashant, Yu, Hui, Harding, Ian, Briem, Stearne, Byrne, Michael, Faraone, Alyse, Friend, Stephen, Huth, Olivia, Iwamoto, Naoki, Kawamoto, Tomomi, Kumarasamy, Jayakanthan, Lamattina, Anthony, Longo, Kenneth, McCarthy, Leah, McGlynn, Andrew, Molski, Allison, Pan, Qianli, Pu, Tom, Purcell-Estabrook, Erin, Rossi, Jeff, Standley, Stephany, Thomas, Carina, Walen, Alexandra, Yang, Hailin, Kandasamy, Pachamuthu, Vargeese, Chandra

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Metabolically Stable Anomeric Linkages Containing GalNAc–siRNA Conjugates: An Interplay among ASGPR, Glycosidase, and RISC Pathways von Kandasamy, Pachamuthu, Mori, Shohei, Matsuda, Shigeo, Erande, Namrata, Datta, Dhrubajyoti, Willoughby, Jennifer L. S., Taneja, Nate, O’Shea, Jonathan, Bisbe, Anna, Manoharan, Rajar M., Yucius, Kristina, Nguyen, Tuyen, Indrakanti, Ramesh, Gupta, Swati, Gilbert, Jason A., Racie, Tim, Chan, Amy, Liu, Ju, Hutabarat, Renta, Nair, Jayaprakash K., Charisse, Klaus, Maier, Martin A., Rajeev, Kallanthottathil G., Egli, Martin, Manoharan, Muthiah

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Control of backbone chemistry and chirality boost oligonucleotide splice switching activity von Kandasamy, Pachamuthu, McClorey, Graham, Shimizu, Mamoru, Kothari, Nayantara, Alam, Rowshon, Iwamoto, Naoki, Kumarasamy, Jayakanthan, Bommineni, Gopal R, Bezigian, Adam, Chivatakarn, Onanong, Butler, David C D, Byrne, Michael, Chwalenia, Katarzyna, Davies, Kay E, Desai, Jigar, Shelke, Juili Dilip, Durbin, Ann F, Ellerington, Ruth, Edwards, Ben, Godfrey, Jack, Hoss, Andrew, Liu, Fangjun, Longo, Kenneth, Lu, Genliang, Marappan, Subramanian, Oieni, Jacopo, Paik, Ik-Hyeon, Estabrook, Erin Purcell, Shivalila, Chikdu, Tischbein, Maeve, Kawamoto, Tomomi, Rinaldi, Carlo, Rajão-Saraiva, Joana, Tripathi, Snehlata, Yang, Hailin, Yin, Yuan, Zhao, Xiansi, Zhou, Cong, Zhang, Jason, Apponi, Luciano, Wood, Matthew J A, Vargeese, Chandra

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FRI-151 RNA editing for the treatment of Alpha-1 antitrypsin deficiency von Monian, Prashant, Lu, Genliang, Shivalila, Chikdu, Bowman, Keith, Bylsma, Marissa, Byrne, Michael, Cannon, Megan, Desai, Jigar, Faraone, Alyse, Favaloro, Frank, Ghosh, Anamitra, Godfrey, Jack, Hernandez, Nidia, Huth, Olivia, Iwamoto, Naoki, Kawamoto, Tomomi, Kumarasamy, Jayakanthan, Kandasamy, Pachamuthu, Lamattina, Anthony, Lindsey, Amber, Liu, Fangjun, Looby, Richard, Luu, Khoa, Metterville, Jake, Paik, Ik-Hyeon, Pan, Qianli, Purcell-Estabrook, Erin, Rheinhardt, Jeanette, Shimizu, Mamoru, Singh, Kuldeep, Standley, Stephany, Thomas, Carina, Tripathi, Snehlata, Yang, Hailin, Yordanoff, Ryan, Yin, Benny, Yu, Hui, Caracta, Cynthia, Narayanan, Padma, Giangrande, Paloma, Vargeese, Chandra

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Quantitative Measurement of Cytosolic and Nuclear Penetration of Oligonucleotide Therapeutics von Deprey, Kirsten, Batistatou, Nefeli, Debets, Marjoke F, Godfrey, Jack, VanderWall, Kirstin B, Miles, Rebecca R, Shehaj, Livia, Guo, Jiaxing, Andreucci, Amy, Kandasamy, Pachamuthu, Lu, Genliang, Shimizu, Mamoru, Vargeese, Chandra, Kritzer, Joshua A

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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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WVE‐005, a stereopure antisense oligonucleotide for MAPT silencing in tauopathies von DaleMaguire, ElenaAbbie, Guo, Lankai, Byrne, Michael, Cannon, Megan, Taborn, Kris, Prakasha, Priyanka Shiva, Longo, Kenneth, Iwamoto, Naoki, Kandasamy, Pachamuthu, Shimizu, Mamoru, Shum, Pochi, Ranade, Ashwini, Iyer, Raghuvaran, Bowman, Keith, Yang, Hailin, Yin, Yuan Benny, Liu, Fangjun, Mohapatra, Susovan, Macovei, Lilia, Patil, Saurabh, Lansita, Janice, Hu, Xiao Shelley, Smith, Karen, Goyal, Jaya, Narayanan, Padma, Rhodes, Ken, Panzara, 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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Stereopure Antisense Oligonucleotides Preferentially Knockdown G4C2 Repeat-Containing C9ORF72 Transcripts: A Potential Therapeutic Approach for the Treatment of Amyotrophic Lateral... von Liu, Yuanjing, Dodart, Jean-Cosme, Tran, Helene, Berkovitch, Shaunna, Byrne, Michael, Durbin, Ann, Iwamoto, Naoki, Jang, Hyun Gyung, Kandasamy, Pachamuthu, Mohapatra, Susovan, Yang, Hailin, Yin, Yuan, Zhang, Jason, Zhong, Zhong, Vargeese, Chandra, Brown, Robert

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Stereochemistry Enhances Potency, Efficacy, and Durability of Malat1 Antisense Oligonucleotides In Vitro and In Vivo in Multiple Species von Byrne, Michael, Vathipadiekal, Vinod, Apponi, Luciano, Iwamoto, Naoki, Kandasamy, Pachamuthu, Longo, Kenneth, Liu, Fangjun, Looby, Richard, Norwood, Lauren, Shah, Anee, Shelke, Juili Dilip, Shivalila, Chikdu, Yang, Hailin, Yin, Yuan, Guo, Lankai, Bowman, Keith, Vargeese, Chandra

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O4‐05‐02: THE INVESTIGATIONAL STEREOPURE ANTISENSE OLIGONUCLEOTIDE WVE‐3972‐01 PREFERENTIALLY REDUCES G4C2 REPEAT‐CONTAINING C9ORF72 TRANSCRIPTS: A POTENTIAL THERAPEUTIC APPROACH F... von Dodart, Jean-Cosme, Liu, Yuanjing, Tran, Helene, Berkovitch, Shaunna, Byrne, Michael, Durbin, Ann, Iwamoto, Naoki, Jang, Hyun Gyung, Kandasamy, Pachamuthu, Liu, Fangjun, Mohapatra, Susovan, Yang, Hailin, Yin, Yuan, Zhang, Jason, Zhong, Zhong, Vargeese, Chandra, Brown, Robert H.

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