biostudies-literatureWang CMinistry of Science and Technology of ChinaMinistry of Science and Technology of the People's Republic of ChinaNatural Science Foundation of Zhejiang ProvinceMinistry of Science and Technology of the People's Republic of ChinaNational Natural Science Foundation of Chinae2411972https://www.ebi.ac.uk/biostudies/studies/S-EPMC11904963biostudies-literatureUnknown12(10)Fragile X syndrome (FXS) is an inherited neurodevelopmental disorder characterized by a range of clinical manifestations with no effective treatment strategy to date. Here, transplantation of GABAergic precursor cells from the medial ganglionic eminence (MGE) is demonstrated to significantly improve cognitive performance in Fmr1 knockout (KO) mice. Within the hippocampus of Fmr1-KO mice, MGE-derived cells from wild-type donor mice survive, migrate, differentiate into functionally mature interneurons, and form inhibitory synaptic connections with host pyramidal neurons. MGE cell transplantation restores Ras-PKB signaling in pyramidal neurons, enhances AMPA receptor trafficking, rescues synaptic plasticity, and corrects abnormal hippocampal neural oscillations. These findings highlight the pAdvanced science (Weinheim, Baden-Wurttemberg, Germany)GABAergic Progenitor Cell Graft Rescues Cognitive Deficits in Fragile X Syndrome Mice.PMC119049633220062032170976816250062023YFE020680032000692LZ24C090003LY21C09000331820103005Zhou LLiu JWang CLu XJWang LSu LDYang WBian YPYe LYWang XTChen WLiu JYWang ZXShen YfalseGABAergic Progenitor Cell Graft Rescues Cognitive Deficits in Fragile X Syndrome Mice.Fragile X syndrome (FXS) is an inherited neurodevelopmental disorder characterized by a range of clinical manifestations with no effective treatment strategy to date. Here, transplantation of GABAergic precursor cells from the medial ganglionic eminence (MGE) is demonstrated to significantly improve cognitive performance in Fmr1 knockout (KO) mice. Within the hippocampus of Fmr1-KO mice, MGE-derived cells from wild-type donor mice survive, migrate, differentiate into functionally mature interneurons, and form inhibitory synaptic connections with host pyramidal neurons. MGE cell transplantation restores Ras-PKB signaling in pyramidal neurons, enhances AMPA receptor trafficking, rescues synaptic plasticity, and corrects abnormal hippocampal neural oscillations. These findings highlight the p2025-01-01T00:00:00Z2025 Mar2026-05-29T17:03:55.302Z2025-04-04T00:32:03.413ZS-EPMC119049633982353410.1002/advs.202411972