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Empty spiracles homeobox 1 (EMX1) is a member of the homeobox family of transcription factors, predominantly expressed in the developing dorsal telencephalon of the brain. It binds double-stranded DNA in a sequence-specific manner and is centrally involved in the regulation of early forebrain development, neuronal differentiation, determination of cortical identity, and neural progenitor cell biology. EMX1, together with its paralog EMX2, modulates gene programs in corticogenesis, neuronal migration, and cell fate specification[1][2][3][4][5]. In knockout animal models, loss of EMX1 leads to selective forebrain abnormalities (notably, agenesis of the corpus callosum and reduced hippocampal size), but does not cause global cortical malformations[1][4]. Emerging data suggests a tumor suppressor role in some sarcomas via repression of stem cell–related transcriptional programs[3]. At present, EMX1 is not a pharmacological drug target and has no established associated clinical biomarkers or direct safety concerns[2][5].
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