The fetal hydrops–associated single-residue mutation L322P disrupts mechanical but not chemical activation of the PIEZO1 ion channel

J Jinghui Jiang (State Key Laboratory of Membrane Biology, New Cornerstone Science Laboratory, Tsinghua-Peking Center for Life Sciences, Beijing Frontier Research Center of Biological Structure, IDG/McGovern Institute for Brain Research, School of Pharmaceutical Sciences, Tsinghua University) W Wei Guo X Xudong Chen (Ministry of Education Key Laboratory of Protein Science, Tsinghua-Peking Center for Life Sciences, Beijing Advanced Innovation Center for Structural Biology, School of Life Sciences, Tsinghua University) Q Qijing He (State Key Laboratory of Membrane Biology, New Cornerstone Science Laboratory, Tsinghua-Peking Center for Life Sciences, Beijing Frontier Research Center of Biological Structure, IDG/McGovern Institute for Brain Research, School of Pharmaceutical Sciences, Tsinghua University) Y Yubo Wang (Department of Chemistry and the Waterloo Institute for Nanotechnology) X Xiaohui Zhu (Jiangsu Key Laboratory of New Energy Devices & Interface Science, School of Chemistry and Materials Science) L Liying Yan (State Key Laboratory of Female Fertility Promotion, Center for Reproductive Medicine, Department of Obstetrics and Gynecology, Peking University Third Hospital) J Jie Qiao (State Key Laboratory of Female Fertility Promotion, Center for Reproductive Medicine, Department of Obstetrics and Gynecology, Peking University Third Hospital) B Bailong Xiao (State Key Laboratory of Membrane Biology, New Cornerstone Science Laboratory, Tsinghua-Peking Center for Life Sciences, Beijing Frontier Research Center of Biological Structure, IDG/McGovern Institute for Brain Research, School of Pharmaceutical Sciences, Tsinghua University)

Abstract

The mechanically activated PIEZO1 ion channel is genetically linked to numerous physiological and pathophysiological processes. For example, deleting PIEZO1 in mice leads to defective lymphatic vessel development, while nonsense mutations in humans are associated with autosomal recessive generalized lymphatic dysplasia (GLD) and nonimmune hydrops fetalis. However, it remains unclear whether PIEZO1-dependent biological processes are directly mediated by its intrinsic mechanosensitivity. Here, we identified a human fetal hydrops–associated single-residue mutation, L322P (corresponding to L329P in mouse PIEZO1). The mutant failed to show mechanically activated currents in response to poking or stretch of the cell membrane, but preserved normal plasma membrane expression and responsiveness to its chemical activators such as Yoda1 and Jedi1. Remarkably, the mechanical response of the mutant can be restored by Yoda1. These findings demonstrate a direct link between the loss of PIEZO1’s mechanosensitivity and the pathophysiological phenotype of fetal hydrops and raise the therapeutic potential of using PIEZO1 chemical activators to restore the mechanosensitivity of PIEZO1 missense mutants that are associated with genetic diseases such as GLD and hydrops fetalis.

Article Details

Volume / Issue Vol. 122, Issue 33
Published August 19, 2025
ISSN 0027-8424
Publisher National Academy of Sciences

Authors (9)

J

Jinghui Jiang

State Key Laboratory of Membrane Biology, New Cornerstone Science Laboratory, Tsinghua-Peking Center for Life Sciences, Beijing Frontier Research Center of Biological Structure, IDG/McGovern Institute for Brain Research, School of Pharmaceutical Sciences, Tsinghua University

W

Wei Guo

X

Xudong Chen

Ministry of Education Key Laboratory of Protein Science, Tsinghua-Peking Center for Life Sciences, Beijing Advanced Innovation Center for Structural Biology, School of Life Sciences, Tsinghua University

Q

Qijing He

State Key Laboratory of Membrane Biology, New Cornerstone Science Laboratory, Tsinghua-Peking Center for Life Sciences, Beijing Frontier Research Center of Biological Structure, IDG/McGovern Institute for Brain Research, School of Pharmaceutical Sciences, Tsinghua University

Y

Yubo Wang

Department of Chemistry and the Waterloo Institute for Nanotechnology

X

Xiaohui Zhu

Jiangsu Key Laboratory of New Energy Devices & Interface Science, School of Chemistry and Materials Science

L

Liying Yan

State Key Laboratory of Female Fertility Promotion, Center for Reproductive Medicine, Department of Obstetrics and Gynecology, Peking University Third Hospital

J

Jie Qiao

State Key Laboratory of Female Fertility Promotion, Center for Reproductive Medicine, Department of Obstetrics and Gynecology, Peking University Third Hospital

B

Bailong Xiao

State Key Laboratory of Membrane Biology, New Cornerstone Science Laboratory, Tsinghua-Peking Center for Life Sciences, Beijing Frontier Research Center of Biological Structure, IDG/McGovern Institute for Brain Research, School of Pharmaceutical Sciences, Tsinghua University