Antisense oligonucleotide therapeutic approach for Timothy syndrome

Xiaoyu Chen, Fikri Birey, Min-Yin Li, Omer Revah, Rebecca Levy, Mayuri Vijay Thete, Noah Reis, Konstantin Kaganovsky, Massimo Onesto, Noriaki Sakai, Zuzana Hudacova, Jin Hao, Xiangling Meng, Seiji Nishino, John Huguenard, Sergiu P Pașca