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Am J Hum Genet ; 111(10): 2232-2252, 2024 Oct 03.
Artículo en Inglés | MEDLINE | ID: mdl-39226899

RESUMEN

The BAF chromatin remodeler regulates lineage commitment including cranial neural crest cell (CNCC) specification. Variants in BAF subunits cause Coffin-Siris syndrome (CSS), a congenital disorder characterized by coarse craniofacial features and intellectual disability. Approximately 50% of individuals with CSS harbor variants in one of the mutually exclusive BAF subunits, ARID1A/ARID1B. While Arid1a deletion in mouse neural crest causes severe craniofacial phenotypes, little is known about the role of ARID1A in CNCC specification. Using CSS-patient-derived ARID1A+/- induced pluripotent stem cells to model CNCC specification, we discovered that ARID1A-haploinsufficiency impairs epithelial-to-mesenchymal transition (EMT), a process necessary for CNCC delamination and migration from the neural tube. Furthermore, wild-type ARID1A-BAF regulates enhancers associated with EMT genes. ARID1A-BAF binding at these enhancers is impaired in heterozygotes while binding at promoters is unaffected. At the sequence level, these EMT enhancers contain binding motifs for ZIC2, and ZIC2 binding at these sites is ARID1A-dependent. When excluded from EMT enhancers, ZIC2 relocates to neuronal enhancers, triggering aberrant neuronal gene activation. In mice, deletion of Zic2 impairs NCC delamination, while ZIC2 overexpression in chick embryos at post-migratory neural crest stages elicits ectopic delamination from the neural tube. These findings reveal an essential ARID1A-ZIC2 axis essential for EMT and CNCC delamination.


Asunto(s)
Proteínas de Unión al ADN , Transición Epitelial-Mesenquimal , Cara , Deformidades Congénitas de la Mano , Discapacidad Intelectual , Micrognatismo , Cuello , Cresta Neural , Factores de Transcripción , Cresta Neural/metabolismo , Factores de Transcripción/genética , Factores de Transcripción/metabolismo , Transición Epitelial-Mesenquimal/genética , Proteínas de Unión al ADN/genética , Proteínas de Unión al ADN/metabolismo , Humanos , Discapacidad Intelectual/genética , Micrognatismo/genética , Animales , Cara/anomalías , Cara/embriología , Deformidades Congénitas de la Mano/genética , Deformidades Congénitas de la Mano/patología , Cuello/anomalías , Cuello/embriología , Ratones , Proteínas Nucleares/genética , Proteínas Nucleares/metabolismo , Células Madre Pluripotentes Inducidas/metabolismo , Células Madre Pluripotentes Inducidas/citología , Haploinsuficiencia , Elementos de Facilitación Genéticos/genética , Deformidades Congénitas del Pie/genética , Deformidades Congénitas del Pie/patología , Regulación del Desarrollo de la Expresión Génica , Anomalías Múltiples
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