Target intelligence / Profile preview

Gon-4-like protein (GON4L)

Target
GON4L
Molecular classification
Transcription co-repressor, Transcription factor regulator, YY1-associated transcriptional regulator, Chromatin-associated protein
01

Overview

Gon-4-like protein (GON4L) is a nuclear protein encoded by the GON4L gene in humans and other vertebrates, highly conserved among species[6][1]. It functions primarily as a transcriptional co-repressor, forming large nuclear complexes that include the transcription factor YY1, co-repressor SIN3A, and histone deacetylase HDAC1, thereby regulating gene expression through chromatin modification and gene repression[2][3]. GON4L is essential for B cell differentiation and broader developmental processes including hematopoiesis and embryogenesis[2][1][7]. It contains specific conserved structural domains such as a paired amphipathic helix (PAH), myb-like domain, and a SANT domain, which mediate protein-protein interactions critical for its functions[2][5]. Mutations in GON4L are associated with developmental syndromes such as Li-Takada-Miyake syndrome, muscle and neurodevelopmental disorders, but it is not currently considered a druggable target or receptor, and no small molecule inhibitors or clinical therapeutics are directed at GON4L as of now[1][4][6].

Other names
Gon-4 likeGON4LGON4KIAA1606FLJ20203YARPGON-4 homologYY1AP related proteinLTMS2610100B20RikYY1AP-related protein1gon-4 homolog
02

Biological functions

Transcriptional repressionNegative regulation of DNA-templated transcriptionB cell developmentChromatin binding
03

Disease associations

Autoimmune disease (e.g., Li-Takada-Miyake syndrome)Muscle diseases (e.g., Oculopharyngodistal myopathy 1)Neurodevelopmental disorders (e.g., Autism spectrum disorder)
04

Safety considerations

Loss-of-function mutations associated with developmental disorders and autoimmunity

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