Increased FAN1 expression by mRNA-LNP attenuates CAG repeat expansion in Huntington patients' iPSC-derived astrocytes
Yung‐Chih Cheng, Gosia Nocula-Lugowska, Julita A Ramirez, Xiaoyu Fan, Fang Jin, Zhihua Jiang, Eric M. Bennett, Jin Li, David E. Hokanson, Sneha Grandhi, Michelle Chen, Congsheng Cheng, Guan-Yu Lin, Laura Lin, Christopher S. Lepsy, , Shian-Huey Chiang
The paper shows that increasing FAN1 expression in HD patient astrocytes reduces CAG repeat expansion using mRNA-LNP.
bioRxiv · Nov 24, 2023 · PreprintNeuroscience