Rescuing Neurodevelopmental Deficits in AMPA Receptor Gain-of-Function Mutant
Chih-Ming Chen, Yu-Min Huang, Chih-Ching Chung, Richard C. Johnson, Cheng-Ya Tsai, Yuhan Chen, Han L. Tan, Fu-Yun Hsiao, Richard L. Huganir, Shu‐Ling Chiu
This study identifies a gain-of-function mutation in GRIA1 causing neurodevelopmental deficits and shows that an antisense oligonucleotide can prevent pathology and behavioral issues.
bioRxiv · Dec 31, 2025 · PreprintNeuroscience