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Article

GM1 Oligosaccharide Ameliorates Rett Syndrome Phenotypes In Vitro and In Vivo via Trk Receptor Activation.

International journal of molecular sciences - 28 Oct 2024

Fazzari Maria, Lunghi Giulia, Carsana Emma Veronica, Valsecchi Manuela, Spiombi Eleonora, Breccia Martina, Casati Silvia Rosanna, Pedretti Silvia, Mitro Nico, Mauri Laura, Ciampa Maria Grazia, Sonnino Sandro, Landsberger Nicoletta, Frasca Angelisa, Chiricozzi Elena

Abstract excerpt

Rett syndrome (RTT) is a severe neurodevelopmental disorder primarily caused by mutations in the methyl-CpG binding protein 2 (MECP2) gene. Despite advancements in research, no cure exists due to an incomplete understanding of the molecular effects of MeCP2 deficiency. Previous studies have identified impaired tropomyosin receptor kinase (Trk) neurotrophin (NTP) signaling and mitochondrial redox imbalances as key...

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Identifiers

PMID: 39519108
DOI: 10.3390/ijms252111555
PMCID: PMC11547101

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