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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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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Modulation of Macrophage Functional Polarity towards Anti-Inflammatory Phenotype with Plasmid DNA Delivery in CD44 Targeting Hyaluronic Acid Nanoparticles von Tran, Thanh-Huyen, Rastogi, Ruchir, Shelke, Juili, Amiji, Mansoor M.

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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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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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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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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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