Antisense oligonucleotide therapy in an individual with KIF1A-associated neurological disorder
Ziegler, A., Carroll, J., Bain, J.M., Sands, T.T., Fee, R.J., Uher. D., Kanner, C.H., Montes, J., Glass, S., Douville, J., Mignon, L., Gleeson, J.G., Crooke, S.T., Chung, W.K.
