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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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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Potent and persistent in vivo anti-HBV activity of chemically modified siRNAs von Morrissey, David V, Lockridge, Jennifer A, Shaw, Lucinda, Blanchard, Karin, Jensen, Kristi, Breen, Wendy, Hartsough, Kimberly, Machemer, Lynn, Radka, Susan, Jadhav, Vasant, Vaish, Narendra, Zinnen, Shawn, Vargeese, Chandra, Bowman, Keith, Shaffer, Chris S, Jeffs, Lloyd B, Judge, Adam, MacLachlan, Ian, Polisky, Barry

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Preclinical evaluation of WVE-004, aninvestigational stereopure oligonucleotide forthe treatment of C9orf72 -associated ALS or FTD von Liu, Yuanjing, Andreucci, Amy, Iwamoto, Naoki, Yin, Yuan, Yang, Hailin, Liu, Fangjun, Bulychev, Alexey, Hu, Xiao Shelley, Lin, Xuena, Lamore, Sarah, Patil, Saurabh, Mohapatra, Susovan, Purcell-Estabrook, Erin, Taborn, Kristin, Dale, Elena, Vargeese, Chandra

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Activity of stabilized short interfering RNA in a mouse model of hepatitis B virus replication von MORRISSEY, David V, BLANCHARD, Karin, POLISKY, Barry A, ZINNEN, Shawn, SHAW, Lucinda, JENSEN, Kristi, LOCKRIDGE, Jennifer A, DICKINSON, Brent, MCSWIGGEN, James A, VARGEESE, Chandra, BOWMAN, Keith, SHAFFER, Chris S

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Biodistribution and metabolism studies of lipid nanoparticle-formulated internally [3H]-labeled siRNA in mice von Christensen, Jesper, Litherland, Karine, Faller, Thomas, van de Kerkhof, Esther, Natt, François, Hunziker, Jürg, Boos, Julien, Beuvink, Iwan, Bowman, Keith, Baryza, Jeremy, Beverly, Mike, Vargeese, Chandra, Heudi, Olivier, Stoeckli, Markus, Krauser, Joel, Swart, Piet

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Novel liver-specific TORC2 siRNA corrects hyperglycemia in rodent models of type 2 diabetes von Saberi, Maziyar, Bjelica, David, Schenk, Simon, Imamura, Takeshi, Bandyopadhyay, Gautam, Li, Pingping, Jadhar, Vasant, Vargeese, Chandra, Wang, Weimin, Bowman, Keith, Zhang, Ye, Polisky, Barry, Olefsky, Jerrold M

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Investigational Assay for Haplotype Phasing of the Huntingtin Gene von Svrzikapa, Nenad, Longo, Kenneth A., Prasad, Nripesh, Boyanapalli, Ramakrishna, Brown, Jeffrey M., Dorset, Daniel, Yourstone, Scott, Powers, Jason, Levy, Shawn E., Morris, Aaron J., Vargeese, Chandra, Goyal, Jaya

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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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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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Intimal Hyperplasia Recurs After Removal of PDGF-AB and -BB Inhibition in the Rat Carotid Artery Injury Model von Leppänen, Olli, Janjic, Nebojsa, Carlsson, Mari-Anne, Pietras, Kristian, Levin, Max, Vargeese, Chandra, Green, Louis S, Bergqvist, David, Östman, Arne, Heldin, Carl-Henrik

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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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WVE-004, an investigational stereopure antisense oligonucleotide for the treatment of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) (2302) von Liu, Yuanjing, Andreucci, Amy, Iwamoto, Naoki, Yin, Yuan, Yang, Hailin, Liu, Fangjun, Patil, Saurabh, Mohapatra, Susovan, Purcell-Estabrook, Erin, Taborn, Kristin, Dale, Elena, Vargeese, Chandra

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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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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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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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