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Impact of guanidine-containing backbone linkages on stereopure antisense oligonucleotides in the CNS
Autor:
Pachamuthu Kandasamy, Yuanjing Liu, Vincent Aduda, Sandheep Akare, Rowshon Alam, Amy Andreucci, David Boulay, Keith Bowman, Michael Byrne, Megan Cannon, Onanong Chivatakarn, Juili Dilip Shelke, Naoki Iwamoto, Tomomi Kawamoto, Jayakanthan Kumarasamy, Sarah Lamore, Muriel Lemaitre, Xuena Lin, Kenneth Longo, Richard Looby, Subramanian Marappan, Jake Metterville, Susovan Mohapatra, Bridget Newman, Ik-Hyeon Paik, Saurabh Patil, Erin Purcell-Estabrook, Mamoru Shimizu, Pochi Shum, Stephany Standley, Kris Taborn, Snehlata Tripathi, Hailin Yang, Yuan Yin, Xiansi Zhao, Elena Dale, Chandra Vargeese
Publikováno v:
Nucleic Acids Research. 50:5401-5423
Attaining sufficient tissue exposure at the site of action to achieve the desired pharmacodynamic effect on a target is an important determinant for any drug discovery program, and this can be particularly challenging for oligonucleotides in deep tis