Antisense oligonucleotides increase Scn1a expression and reduce seizures and SUDEP incidence in a mouse model of Dravet syndrome

著者: Han Zhou, Chunling Chen, Anne Christiansen, Sophina Ji, Qian Lin, Charles Anumonwo, Chanté Liu, Steven C. Leiser, Meena Meena, Isabel Aznarez, Gene Liau, Lori L. Isom - Science Translational Medicine 2020 被引用: 336

1.1 protein was confirmed in brains of treated mice. Our results suggest that TANGO may provide a unique, gene-specific approach for the treatment of DS.