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Article

Modeling Rett Syndrome with Human Pluripotent Stem Cells: Mechanistic Outcomes and Future Clinical Perspectives.

International journal of molecular sciences - 3 Apr 2021

Gomes Ana Rita, Fernandes Tiago G, Cabral Joaquim M S, Diogo Maria Margarida

Abstract excerpt

Rett syndrome (RTT) is a neurodevelopmental disorder caused by mutations in the gene encoding the methyl-CpG-binding protein 2 (MeCP2). Among many different roles, MeCP2 has a high phenotypic impact during the different stages of brain development. Thus, it is essential to intensively investigate the function of MeCP2, and its regulated targets, to better understand the mechanisms of the disease and inspire the...

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Identifiers

PMID: 33916879
DOI: 10.3390/ijms22073751
PMCID: PMC8038474

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