Antisense oligonucleotide therapy in an individual with KIF1A-associated neurological disorder
Alban Ziegler, Joanne Carroll, Jennifer Bain, Tristan T. Sands, Robert J. Fee, David Uher, Cara H. Kanner, Jacqueline Montes, Sarah M. Glass, Julie Douville, Laurence Mignon, Joseph G. Gleeson, Stanley T. Crooke, Wendy K. Chung
Topics & Concepts
AlleleOligonucleotideMedicineDiseaseBiologyBioinformaticsGeneticsGeneInternal medicineGenomics and Rare DiseasesChromosomal and Genetic VariationsGenomic variations and chromosomal abnormalities