Antisense oligonucleotides to KIF1A polymorphisms expand targets and rescue patient-derived neurons in vitro
- Michael V. Zuccaro
- Randee E. Young
- Jiangyuan Hu
- Patricia Lanzano
- Ekaterina Semenova
- Xiaoqi Lin
- Alicja Skowronski
- Yufeng Shen
- Tae Hyun Kim
- Danny E. Miller
- Noelle Germain
- Patrick Sarmiere
- Charles A. LeDuc
- Wendy K. Chung
2026-01-09