Gene therapy using human FMRP isoforms driven by the human FMR1 promoter rescues fragile X syndrome mouse deficits.

Jiang Y, Han L, Meng J, Wang Z, Zhou Y, Yuan H, Xu H, Zhang X, Zhao Y, Lu J, Xu H, Zhang C, Zhang YW
Mol Ther Methods Clin Dev. 2022 Oct 7;27:246-258. doi: 10.1016/j.omtm.2022.10.002. eCollection 2022 Dec 8. PubMed Article

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