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Purine-rich element-binding protein A (PURA) is a highly conserved, sequence-specific single-stranded DNA- and RNA-binding protein best known for its regulatory roles in gene transcription and mRNA localization. It binds preferentially to purine-rich single-stranded DNA or RNA and is involved in the unwinding of double-stranded nucleic acids in an ATP-independent manner, which is critical for nucleic acid structural remodeling. This protein is a member of the ancient "Pur" family, with a structural fold known as a Whirly-like domain, mediating both nucleic acid binding and protein-protein interactions. PURA plays diverse roles in neurodevelopment, and mutations in the PURA gene cause PURA syndrome, a neurodevelopmental disorder marked by hypotonia, developmental delay, and other neurological deficits. PURA has also been implicated in viral replication, such as HIV, by binding viral RNAs. There are no established pharmacological agents that target PURA directly, although it is a potential therapeutic and diagnostic target in neurological and viral diseases.
No drugs approved that act through direct modulation; thus, mechanism of drug action on this target is not established.
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