Antisense oligonucleotide therapy in an individual with KIF1A-associated neurological disorder

作者：Alban C. Ziegler, Joanne M. Carroll, Jennifer Bain, Tristan T. Sands, Robert J. Fee, David T. Uher, Cara H. Kanner, Jacqueline Montes, Sarah M. Glass, Julie Douville, Laurence Mignon, Joseph G. Gleeson, Stanley T. Crooke, Wendy K. Chung · 发表于：Nature Medicine · 年份：2024 · DOI：10.1038/s41591-024-03197-y · 被引用次数：60 · 研究领域：Genomics and Rare Diseases、Chromosomal and Genetic Variations、Genomic variations and chromosomal abnormalities