Stereochemistry Enhances Potency, Efficacy, and Durability of Malat1 Antisense Oligonucleotides In Vitro and In Vivo in Multiple Species

por 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
Publicado 2021

Variant-selective stereopure oligonucleotides protect against pathologies associated with C9orf72-repeat expansion in preclinical models

por 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.
Publicado 2021

Impact of stereopure chimeric backbone chemistries on the potency and durability of gene silencing by RNA interference

por 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
Publicado 2023

Impact of guanidine-containing backbone linkages on stereopure antisense oligonucleotides in the CNS

por 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
Publicado 2022

Control of backbone chemistry and chirality boost oligonucleotide splice switching activity

por 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
Publicado 2022