Antisense oligonucleotide therapy in an individual with KIF1A-associated neurological disorder
反义寡核苷酸疗法治疗KIF1A相关神经系统疾病患者
期刊:Nature Medicine
影响因子:50
doi:10.1038/s41591-024-03197-y
Ziegler, Alban; Carroll, Joanne; Bain, Jennifer M; Sands, Tristan T; Fee, Robert J; Uher, David; Kanner, Cara H; Montes, Jacqueline; Glass, Sarah; Douville, Julie; Mignon, Laurence; Gleeson, Joseph G; Crooke, Stanley T; Chung, Wendy K