A framework for individualized splice-switching oligonucleotide therapy

Kim, J; Woo, S; de Gusmao, CM; Zhao, BX; Chin, DH; DiDonato, RL; Nguyen, MA; Nakayama, T; Hu, CA; Soucy, A; Kuniholm, A; Thornton, JK; Riccardi, O; Friedman, DA; El Achkar, CM; Dash, Z; Cornelissen, L; Donado, C; Faour, KNW; Bush, LW; Suslovitch, V; Lentucci, C; Park, PJ; Lee, EA; Al Patterson; Philippakis, AA; Margus, B; Berde, CB; Yu, TW

Kim, J (通讯作者),Korea Adv Inst Sci & Technol KAIST, Grad Sch Med Sci & Engn, Daejeon, South Korea.;Kim, J (通讯作者),Korea Adv Inst Sci & Technol KAIST, Biomed Res Ctr, Daejeon, South Korea.;Kim, J (通讯作者),Korea Adv Inst Sci & Technol KAIST, KI Hlth Sci & Technol, Daejeon, South Korea.;Kim, J (通讯作者),Korea Adv Inst Sci & Technol KAIST, Ctr Epidem Preparedness, Daejeon, South Korea.;Yu, TW (通讯作者),Boston Childrens Hosp, Div Genet & Genom, Boston, MA 02115 USA.;Yu, TW (通讯作者),Boston Childrens Hosp, Manton Ctr Orphan Dis Res, Boston, MA 02115 USA.;Yu, TW (通讯作者),Boston Childrens Hosp, Dept Pediat, Boston, MA 02115 USA.;Yu, TW (通讯作者),Broad Inst MIT & Harvard, Program Med & Populat Genet, Cambridge, MA 02142 USA.;Yu, TW (通讯作者),Harvard Med Sch, Boston, MA 02115 USA.

NATURE, 2023; 619 (7971): 828

Abstract

Splice-switching antisense oligonucleotides (ASOs) could be used to treat a subset of individuals with genetic diseases(1), but the systematic identif......

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